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1st International Electronic Conference on Brain Sciences
Part of the International Electronic Conference on Brain Sciences series
10–25 Nov 2020
Neuroscience, Brain Function, Nervous System
- Go to the Sessions
- Event Details
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- Welcome from the Chair
- Webinar | Advances in diagnosis and treatment in MS
- Webinar Content
- Event Calls
- Conference Organizers
- Conference Chairs
- Conference Speakers
- IECBS 2020: Dr. KazuhitoToyooka's presentation
- Sessions
- Instructions for Authors
- List of Accepted Submissions
- List of Authors
- Event Awards
- Sponsors and Partners
- The 1st International Electronic Conference on Brain Sciences (IECBS 2020) - Editor Interview video
- Events in this series
All participants of IECBS2020 are welcome to submit the extended work to the Brain Sciences Special Issue "Selected Papers from the 1st International Electronic Conference on Brain Sciences".
Welcome from the Chair
You are cordially invited to participate in the 1st International Electronic Conference on Brain Sciences. This event aims to bring together researchers working in the field of neuroscience, to present and discuss their recent contributions, without the need for travel. During this e-conference, a live session will also be held on 10 November 2020.
The field of neuroscience is one of the last frontiers in biomedical studies, as many of the details that control brain function are still not well understood. This makes the study of the nervous system very exciting and fast moving. This conference will address a variety of research topics, which reflect some of the current areas of focus. These are organized into seven sessions, and include:
- Clinical Neuroscience
- Cognitive Neuroscience
- Systems Neuroscience
- Molecular and Cellular Neuroscience
- Developmental Neuroscience
- Behavioral Neuroscience
- Environmental Neuroscience
Accepted abstract submissions will be published in the proceedings of the conference, and authors are invited to elaborate their abstracts into full manuscripts that will be considered for publication in Brain Sciences, with a 20% discount on the APC. Brain Sciences is an open access journal from MDPI, in the field of neuroscience. The journal is indexed in the Sciences Citation Indexing Expended (SCIE IF=3.332), Scopus, and other databases. Citations are available in PubMed; full-text archived in PubMed Central (PMC). Please visit the following website for more information.
https://www.mdpi.com/journal/brainsci
Important Dates
• Abstract Submission: 15 September 2020
• Notification of Acceptance: 1st October 2020
• Submission of Full Manuscript to Brain Sciences: 25 October 2020
• Conference Date: 10–25 November 2020
You are welcome both to upload and present your work and to attend the conference completely free of charge.
Please do not hesitate to contact us if you have questions.
Best wishes,
Stephen D. Meriney, PhD
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If you have any questions, please feel free to contact us:
Conference Secretariat
Ms. Amanda Zhang
Ms. Sammi Wang
Ms. Alexis Liu
MDPI Brain Sciences editorial Office, Beijing
E-Mail: iecbs2020@mdpi.com
Webinar | Advances in diagnosis and treatment in MS
Webinar Information
Webinar Date: 10 November 2020
Time: 9 -10:30 am (US Eastern time) | 15:00 p.m.-16:30 (CET)
Webinar ID: 810 1943 7849
Webinar Secretariat: iecbs2020@mdpi.com
It is well accepted that the last decade has been marked by enormous advances in the field of demyelinating diseases and more specifically multiple sclerosis. A lot of research occurred leading to early diagnosis and discovery of new biomarkers that help with treatment decisions. New therapies emerged and moved from the lab to human trials, to pharmacies and eventually to the patient’s home. Nowadays, diagnosis of MS is not a death sentence. It is viewed as chronic disease that can be managed very well and, given successful treatment, patients can enjoy a high quality of life.
During this webinar, we will focus on two fundamental areas of research in MS. The first presentation by Dr. Mark Haacke will discuss a new imaging method, the MICRO Imaging that can image very small vessels in the brain, including both arteries and veins, and subsequently classify vascular abnormalities, MS-specific.
The second presentation by Dr. Robert Lisak will discuss the role exosomes as mediators of immune regulation in MS and will review evidence about the toxicity of exosome- enriched fractions from B-lymphocytes on oligodendrocytes in people with MS.
The following expert researcher will present and speak:
Chair: Dr. Evanthia Bernitsas
Evanthia Triantafylou-Bernitsas, MD joined the Department of Neurology at Wayne State University in 2011. She has completed a neurology residency at Case Western Reserve University and Neuroimmunology Fellowships at the University of Michigan and Baylor College of Medicine. She is the Director of the Multiple Sclerosis Center at Wayne State School of Medicine and an Associate Professor of Neurology.
Her clinical research focuses on aggressive forms of MS, new immunomodulatory therapies and symptomatic interventions. Dr. Bernitsas directs a very active clinical research program and she is the PI on numerous clinical and investigator-initiated trials.
Her laboratory research interests include novel neuroimaging and optical coherence tomography that lead to a better understanding of the disease, pathophysiology and imaging of MS fatigue and the use of biomarkers in neuroprotection and remyelination. Currently, she serves as the Editor-in Chief in the Neuroimaging section of the “Brain Sciences” Journal. She has published extensively and served in several committees and advisory boards.
Speaker: Dr. Robert Lisak
It is our honor to present Dr. Robert Lisak in today’s webinar. Dr. Robert Lisak is the Parker Webber endowed Chair in Neurology at Wayne State School of Medicine, a faculty member at the Neurology Department and a world renowned physician scientist. His career spans several decades, starting first at the University of Pennsylvania, where he was faculty, Vice Chair of Neurology and member of the graduate group in Immunology. Then, Dr. Lisak moved to Detroit and became the Chair of the Neurology Department at the Wayne State School of Medicine for 25 years, neurologist in Chief at Detroit Medical Center and Chief of Neurology at Harper Hospital. During his career he has won prestigious awards, served as Editor in Chief in the Journal of the Neurological Sciences and has had funding from NIH, NMS Society, Muscular Dystrophy Association and Myasthenia Gravis Foundation of America, Guillan- Barre/CIDP Foundation and pharma support for multicenter clinical trials and IIT studies. He has authored or co-authored more than 350 peer reviewed paper or reports. Bob, welcome!
Speaker: Dr. Ewart Mark Haacke
It is our pleasure to present Dr. Ewart Mark Haacke. Dr. E. M. Haacke, PhD is the Director of the MR Research facility and a professor of Radiology and Biomedical Engineering at Wayne State University, Vice Chairman of Biomedical Engineering, a professor of Radiology at Loma Linda University and a Professor of Physics at Case Western Reserve University.
He is a pioneer of the MR angiographic imaging, and he is the first to develop a powerful imaging method, the susceptibility weighted imaging (SWI) that detects micro-hemorrhages and iron. This work later extended to quantitative susceptibility mapping (QSM) that can quantify iron. During his long career, he has served in many committees and won prestigious awards, including the Gold Medal of the International Society for Magnetic Resonance in Medicine. He has more than 40- years experience in the MRI research, trained many residents and fellows, and authored or co-authored more than 300 peer- review papers. Welcome Mark!
Webinar Content
Chair: Dr. Evanthia Bernitsas
Speakers: Dr. Robert Lisak and Dr. Ewart Mark Haacke
Date and time: 10 November 2020, 3:00 PM (CET)
Call for Abstracts
Abstract: A single paragraph of around 200 words maximum. For research articles, abstracts should give a pertinent overview of the work. We strongly encourage authors to use the following style of structured abstracts, but without headings: (1) Background: Place the question addressed in a broad context and highlight the purpose of the study; (2) Methods: Describe briefly the main methods or treatments applied; (3) Results: Summarize the article's main findings; and (4) Conclusions: Indicate the main conclusions or interpretations. The abstract should be an objective representation of the article, it must not contain results which are not presented and substantiated in the main text and should not exaggerate the main conclusions.
Abstract Submission: 15 September 2020You are welcome both to upload and present your work and to attend the conference completely free of charge.
Conference Organizers
MDPI -Brain Sciences
Brain Sciences Editorial Office
MDPI, St. Alban-Anlage 66, 4052 Basel, Switzerland
Brain Sciences (ISSN 2076-3425) is an international peer-reviewed open access journal on neuroscience published monthly online by MDPI. The journal is indexed in the Sciences Citation Indexing Expended (SCIE IF=2.786), Scopus and other databases. Citations available in PubMed, full-text archived in PubMed Central (PMC).
Please visit its website for more information https://www.mdpi.com/journal/brainsci.
brainsci@mdpi.com
Conference Chairs
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Stephen D. Meriney completed his PhD in Physiology/Neuroscience at the University of Connecticut with Dr. Guillermo Pilar in 1986, where he studied the parasympathetic control of intrinsic eye muscles. He then moved to the Jerry Lewis Neuromuscular Research Centre at the University of California, Los Angeles (UCLA) as a postdoctoral fellow, and then became an assistant research physiologist there, with Dr. Alan Grinnell, and studied voltage-gated calcium channels and synaptic mechanisms at the neuromuscular junction. He has been at the University of Pittsburgh since 1993, where he is currently a Professor, and Chair of the Department of Neuroscience, with a secondary appointment in the Department of Psychiatry at the University of Pittsburgh Medical Center. He is currently Editor-in-Chief of the journal Brain Sciences.
Conference Committee
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School of Behavioral and Brain Sciences, The University of Texas at Dallas, USA
research interests: prefrontal cortex; schizophrenia; drug addiction; working memory; catecholamines
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Department of Neurobiology and Anatomy, Drexel University College of Medicine, Philadelphia, Pennsylvania, PA, USA
homepage: neurobio.drexelmed.edu/ToyookaWeb/toyooka.html
research interest:neuronal migration; cortical development; neuronal migration disorders; neurodevelopmental disorders; cerebral cortical malformation; neurite formation; neurite initiation; spine formation
kt469@drexel.edu
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research interest:neuromodulation; synaptic plasticity; neuro-glia interaction; functional restoration; neurodegenerative disorders
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Department of Neurology, Kansas University Medical Center, USA
research interest:Neurodegeneration; Ocular diseases; Structure-Funtion of proteins; Naturally Unfolded Proteins
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School of Biomedical Engineering, Science and Health Systems, Drexel University, USA
research interest:Neuroergonomics; functional neuroimaging, biomedical signal processing; neuroengineering; functional near infrared spectroscopy (fNIRS); electroencephalogram (EEG); brain computer interfaces (BCI); mobile brain/body imaging (MoBI); cogni
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Department of Developmental Psychology, University of Santiago de Compostela, Spain
current research interest are the longitudinal process of cognitive aging, including early signs of cognitive impairment, mild cognitive impairment, subjective cognitive decline, mild behavioral impairment, cognitive frailty, the role of cognitive reserve in early manifestation of the disease, and the development computerized cognitive interventions to prevent dementia and other age-related diseases. Member of the Galician research networks about frailty (http://frailnet.es) and dementia (http://regidem.imaisd.es). Editorial Board member of the journal Geriatrics (http://www.mdpi.com/journal/geriatrics/editors) and Brain Sciences (https://www.mdpi.com/journal/brainsci/editors). Review Expert for the European Commission in Horizon 2020 H3 – E-Health, Well-being and Ageing and for the 2017 and 2019 Calls of the Andalusian Government's Health Department.
research interest:longitudinal process of cognitive aging; early signs of cognitive impairment; Mild Cognitive Impairment and mild dementia; the role of cognitive reserve in early manifestation of the disease; the development computerized cognitive interv
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Faculty at Columbia University Medical Center, Scientist, Division of Analytical Psychopharmacology, Nathan Kline Institute for Psychiatric Research, USA
research interest:cannabinoid receptors; synaptic plasticity; learning and memory; developmental disorders; neurodegeneration; drugs of abuse and psychiatric disorders
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EA4391 Excitabilité Nerveuse & Therapeutique, Université Paris Est Créteil,France,
Neurophysiology department, Henri Mondor Hospital, France
Dr. Samar S. Ayache got her Doctor of Medicine (M.D.) from the Lebanese university in Beirut (2003) and then pursued her residency in Neurology between Lebanon and France (University Paris-Est Creteil, UPEC, 2009). Afterwards, she continued graduate studies in Neurophysiology (UPEC & Lille 2 University, 2010), Neurosciences (M.Sc., UPEC, 2011) and Pathophysiology (Ph.D., UPEC, 2014). Her neurophysiology training includes electromyography, electroencephalography (EEG), evoked potentials and the use of noninvasive brains stimulation (NIBS) techniques –namely magnetic and electric stimulation- in diagnostic and therapeutic purposes. Since 2014, Dr. Ayache is the head of EEG unit at the neurophysiology department in Henri Mondor Hospital, Creteil, France. She is also an associate professor of medical education at UPEC, reviewer in several international journals, and a member of many scientific and medical organizations (i.e., the French-Speaking Society of Clinical Neurophysiology, the French Society of Multiple Sclerosis, and the French Society of Peripheral Nerve). Her research activity led to more than eighty peer-reviewed scientific publications, and more than sixty invited lectures and conference papers. Her featuring topics are the pathophysiological mechanisms underlying pain syndromes, MS manifestations, movement disorders, and the optimization of NIBS effects. Her future projects include the therapeutic implementation of neuro-navigated NIBS and the application of high-resolution EEG- and MRI-coupled NIBS techniques. This aims for a better understanding of the neurophysiological correlates of NIBS and the related changes in brain networks; and may allow selecting the best parameters and the optimal cortical targets to maximize the therapeutic effects of NIBS.
research interest:Multiple sclerosis; MS fatigue; MS pain; neurophysiology; evoked potentials; electroencephalography; Non-invasive brain stimulation
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Section on Gene Structure and Disease, Laboratory of Cell and Molecular Biology, National Institute of Diabetes, Digestive and Kidney Diseases,USA
Dr. Daman Kumari is a Staff Scientist in the Laboratory of Cell and Molecular Biology at the National Institutes of Health. She has made several important contributions to the understanding of FMR1 gene silencing in fragile X syndrome, and her current research is focused on developing treatment strategies based on restoring FMR1 expression. Dr. Kumari received her PhD in Life Sciences from the Jawaharlal Nehru University in New Delhi, India where she developed molecular assays for the differential diagnosis of Duchenne and Becker muscular dystrophy. She completed her postdoctoral training at the National Institutes of Health with Dr. Karen Usdin and was promoted to the Staff Scientist position in 2006. Dr. Kumari serves on the editorial board of Brain Sciences, Genetic Testing and Molecular Biomarkers, and the Journal of Autism and Developmental Research.
research interest:gene expression; stem cells; chromatin biology; biomarkers
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Curtin Neuroscience Laboratory, School of Psychology and Speech Pathology, Curtin University, Australia
Associate Professor Andrea Loftus is a researcher and lecturer in the School of Psychology at Curtin University, Australia. Andrea is the Director of ParkC, a collaborative research group whose primary goal is to improve the quality of life of people affected by Parkinson's Disease (Parkinson’s) by optimizing collaborative research between different research disciplines and Parkinson's communities. Andrea’s research focuses on the identification and management of cognitive symptoms in Parkinson’s, and she has a particular interest in the therapeutic potential of non-invasive brain stimulation for improving cognitive function in Parkinson’s . Andrea has recently published work evaluating the nature of cognitive impairment in Parkinson’s, the relationship between cognition and sleep in Parkinson’s, and the potential of non-invasive brain stimulation for cognitive deficits associated with Parkinson’s.
research interest:parkinson’s disease; mild cognitive impairment; cognition; neurodegeneration; plasticity; brain stimulation
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Sheffield Institute for Translational Neuroscience, Department of Neuroscience, University of Sheffield, UK
Dr Alisdair McNeill is a Senior Clinical Lecturer and Consultant Physician in Clinical Genetics at the University of Sheffield and Sheffield Children's Hospital. His clinical and research interests are inherited neurological disorders affecting children and adults. He uses exome and genome technologies to identify novel disease causing genes, and has identified SOX4, SOX11, MYT1L and SLC12A2 as causes of neurodevelopmental disorders. He also has an interest in studying the earliest phases (prodromal phase) of neurological disorders by using genetically at risk populations such as GBA or 22q11 deletion carriers. In these populations he utilises human movement analysis technology to identify the earliest stages of movement disorders.
research interest:neurogenetics; deep phenotyping; next generation sequencing; qualitative studies; rare disease
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Hillary Wehe is an Assistant Professor of Psychology at Davis & Elkins College in West Virginia. She received her Ph.D. in Cognitive Psychology from Colorado State University, where she was a teaching fellow and the thee-time recipient of Colorado State’s Mark V. Richard Excellence in Teaching Award. Before joining the faculty at Davis & Elkins College, she taught at Georgia State University Armstrong Campus. Dr. Wehe's research focuses on student motivation and learning. Specifically, she explores the constraints that external rewards place on students' personal interest for learning and she works to apply these research interests in the classroom to create a student-centered, active, and engaging environment. To explore these initiatives, she has recently been awarded the West Virginia Behavioral Health Workforce Initiative Grant and a NetVUE Vocation Across the Academy grant.
research interest:motivation; reward; learning; decision-making
Invited Speakers
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Department of Neurobiology and Anatomy, Drexel University College of Medicine, Philadelphia, Pennsylvania, PA, USA.
Dr. Toyooka will making a speech during the e-conference, the speech title is "Adnp shuttling to the cytoplasm by 14-3-3 is essential to promote neuronal morphogenesis and functional cortical connectivity".
Dr. Toyooka is an assistant professor in the Department of Neurobiology & Anatomy at Drexel University College of Medicine. He did postdoctoral fellowships at the University of California San Diego and the University of California San Francisco. He also served on the faculty at Osaka City University School of Medicine in Japan. He was appointed to the faculty in the Department of Neurobiology & Anatomy at the College of Medicine in 2013.
neuronal migration; cortical development; neuronal migration disorders; neurodevelopmental disorders; cerebral cortical malformation; neurite formation; neurite initiation; spine formation
Instructions for Authors
Submissions should be submitted by the authors online by registering at https://iecbs.sciforum.net/, and using the “Submit Abstract” function once logged into system.
- Scholars interested in participating with the conference can submit their abstract (about 200–250 words covering the areas of manuscripts for the proceedings issue) online at this website up to 15 September 2020.
- The Conference Committee will conduct a pre-evaluation, based on the submitted abstract, of whether the contribution from the authors of the abstract will be welcome for the 1st International Electronic Conference on Brain Sciences. All authors will be notified by 1st October 2020 about the acceptance of their abstract.
- If the abstract is accepted for this conference, the author is asked to submit the manuscript optionally along with a PowerPoint and/or video presentation of his/her paper (only PDF), up to the submission deadline of 25 October 2020.
- The conference proceedings papers and presentations will be available at https://iecbs.sciforum.net/ for discussion during the time of the conference, 10–25 November 2020, and will be published in Journal Proceedings.
- The open access journal Brain Sciences will publish a Special Issue of the conference and accepted papers will be published in the proceedings of the conference itself. After the conference, the Conference Committee will select manuscripts that may be included for publication in the Special Issue of the journal Brain Sciences (the submission to the journal is independent from the conference proceedings and will follow the usual process of the journal, including peer-review, APC, etc.).
First page:
- Title
- Full author names
- Affiliations (including full postal address) and authors’ e-mail addresses
- Abstract (200–250 words)
- Keywords
- Introduction
- Methods
- Results and Discussion
- Conclusions
- (Acknowledgements)
- References
Manuscripts should be prepared in MS Word or any other word processor and should be converted to PDF format before submission. The publication format will be PDF. The manuscript should be at least 3 pages (incl. figures, tables, and references) and should not exceed 6 pages.
Presentation Slides
Authors are encouraged to prepare a presentation in PowerPoint or similar software, to be displayed online along with the manuscript. Slides, if available, will be displayed directly in the website using the proprietary slides viewer at Sciforum.net. Slides can be prepared in exactly the same way as for any traditional conference where research results can be presented. Slides should be converted to PDF format before submission so that we can easily and automatically process them for online display.
Video Presentations
Besides their active participation within the forum, authors are also encouraged to submit video presentations. The video should be no longer than 20 minutes and be prepared in one of the following formats:
- MOV
- MPEG4
- MP4
- AVI
- WMV
- MPEGPS
- FLV
Authors that wish to present only a poster, i.e., without a proceedings paper, can do so in section I. Posters will be available on the conference website during and after the event. Similarly to papers presented at the conference, participants will be able to ask questions and make comments about the posters. Posters that are submitted without a paper will not be included in the proceedings of the conference.
Submission: Manuscripts should be submitted online at https://iecbs.sciforum.net by registering and logging in to this website.
Accepted File Formats
- MS Word: Manuscript prepared in MS Word must be converted into a single file before submission. When preparing manuscripts in MS Word, 1st International Electronic Conference on Brain Sciences Microsoft Word template file (see download below) must be used. Please do not insert any graphics (schemes, figures, etc.) into a movable frame which can superimpose the text and make layout very difficult.
- LaTeX: Manuscripts prepared in LaTeX must be collated into one ZIP folder (include all source files and images so that the Conference Secretariat can recompile the submitted PDF). When preparing manuscripts in LaTeX, please use the 1st International Electronic Conference on Brain Sciences LaTeX template files.
1st International Electronic Conference on Brain Sciences Microsoft Word template file and LaTex template file
- Paper Format: A4 paper format, the printing area is 17.5 cm × 26.2 cm. The margins should be 1.75 cm on each side of the paper (top, bottom, left, and right sides).
- Paper Length: The conference proceedings paper should not be longer than 6 pages. The conference manuscript should be as concise as possible.
- Formatting/Style: The paper style of the journal Proceedings should be followed. You may download the template file to prepare your paper (see above). The full titles of the cited papers must be given. Reference numbers should be placed in square brackets [ ], and placed before punctuation; for example [4] or [1–3], and all the references should be listed separately and as the last section at the end of the manuscript.
- Authors List and Affiliation Format: Authors’ full first and last names must be given. Abbreviated middle name(s) can be added. For papers written by various contributors, a corresponding author must be designated. The PubMed/MEDLINE format is used for affiliations: complete street address information including city, zip code, state/province, country, and email address should be added. All authors who contributed significantly to the manuscript (including writing a section) should be listed on the first page of the manuscript, below the title of the article. Other parties who provided only minor contributions should only be listed under Acknowledgments. A minor contribution might be a discussion with the author, reading through the draft of the manuscript, or performing English corrections.
- Figures, Schemes, and Tables: Authors are encouraged to prepare figures and schemes in color. Full color graphics will be published free of charge. Figure and schemes must be numbered (Figure 1, Scheme I, Figure 2, Scheme II, etc.) and an explanatory title must be added. Tables should be inserted into the main text, and numbers and titles for all tables supplied. All table columns should have an explanatory heading. Please supply legends for all figures, schemes, and tables. The legends should be prepared as a separate paragraph of the main text and placed in the main text before a table, figure, or scheme.
Potential Conflicts of Interest
It is the authors’ responsibility to identify and declare any personal circumstances or interests that may be perceived as inappropriately influencing the representation or interpretation of clinical research. If there is no conflict, please state here “The authors declare no conflict of interest”. This should be conveyed in a separate “Conflict of Interest” statement preceding the “Acknowledgments” and “References” sections at the end of the manuscript. Financial support for the study must be fully disclosed under the “Acknowledgments” section.
Copyright
MDPI, the publisher of the Sciforum.net platform, is an open access publisher. We believe that authors should retain the copyright to their scholarly works. Hence, by submitting a Communication paper to this conference, you retain the copyright of your paper, but you grant MDPI the non-exclusive right to publish this paper online on the Sciforum.net platform. This means you can easily submit your paper to any scientific journal at a later stage and transfer the copyright to its publisher (if required by that publisher).
List of accepted submissions (11)
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sciforum-037086 | Exploring inflammatory status in febrile seizures associated with urinary tract infections: a Two-Step cluster approach | , , , |
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Background: Urinary tract infections (UTI) are considered as common facilitating factors along with other infections in triggering febrile seizures (FS). The main purpose of the study is to identify specific patterns of UTIs, using a combination of inflammatory biomarkers, in order to differentiate UTIs from other bacterial diseases associated with FS. Method: This study included a number of 197 distinct FS events, from patients hospitalized in the Sibiu Pediatric Hospital, among which 10.2% were diagnosed with UTIs. Results: In one third of patients with UTIs symptoms were limited to fever and FS. Using Two-Step cluster analysis, a distinct inflammatory pattern has emerged: higher PDW (median value 9,65 fl), P-LCR (median value 14,45%), VTM (median value 10,40 fl), PCR (median value 74,00 g/L) and neutrophil-lymphocyte ratio (median value 3,64), associated mainly (85,7%) with bacterial respiratory infections. UTIs were highly unlikely in the patients with significantly increased PCR values and normal values of platelet indices. Conclusion. Considering the nonspecific clinical picture of UTIs at an early age, in order to optimize the management of FS a fast diagnosis of UTI is mandatory. Our study suggests that analyzing the inflammatory biomarkers interlink (rather than individual parameters) could help identify UTI patients, even when oligosymptomatic. |
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sciforum-037121 | A Novel Retinal Gene Therapy Strategy for Batten Disease and Beyond | , , , , , , , |
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Batten Disease is a fatal, lysosomal storage disorder characterized by cognitive and motor deficits, vision impairments, and seizures. Loss of vision is a hallmark of 10 of the 13 Batten Disease subtypes. Our group has pioneered AAV9 gene therapy treatments achieving widespread transduction of the brain and spinal cord. Two clinical trials are currently ongoing at Nationwide Children’s Hospital delivering this vector via cerebrospinal fluid (CSF) to the brain and spinal cord for treatment of the lethal neurodegenerative aspects of Batten Disease. However, AAV9 transduction of the retina after CSF delivery is limited and there is a critical need for a solution that prevents vision loss and further improves quality of life for Batten Disease patients. Similar to most genetic ocular diseases, photoreceptor degeneration is the most commonly cited pathology in patients. However, recent studies suggest that in some subtypes of Batten Disease, expression must be rescued also within the deeper layers of the retina that are difficult to reach with therapeutic vectors (inner nuclear layer, INL). We performed single cell RNA sequencing of mice and non-human primate retinas in collaboration with Dr. Fischer (OSU) and concluded that Batten Disease vision specific gene therapy needs to target a wide range of cells, including the INL, which is a major challenge for translation of a vision specific therapy to the clinic. We have recently discovered that administration of neuraminidase (NA), a sialidase enzyme, prior to or in combination with AAV9.GFP, drastically increases transduction throughout the murine C57Bl/6 retina including the INL and all the way through to the photoreceptor layer. Our preliminary data indicates GFP expression in almost all, if not all, retinal cell types using this method. Importantly, we have confirmed successful targeting of INL bipolar cells, a notoriously difficult cell-type to transduce, and up to 40% increase in Müller glia transduction. Preliminary histological examination indicates no damage or alterations in retinal integrity. While additional testing in large animal models is required and scheduled to occur in a WT pig model in Autumn 2020, this remarkable discovery suggests that it may now be possible to target every cell-type of the retina with a single AAV vector. This is especially important in regard to treatment of Batten Disease but has many additional implications. Current retinal therapies that require a more invasive subretinal delivery to photoreceptors could now opt for the safer intravitreal delivery strategy. In addition, this strategy would be highly useful in the field of optogenetics, where investigators continue to struggle to find an efficient way to express light-sensitive opsins in cells of the INL to restore sight in individuals that have already lost their photoreceptors. |
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sciforum-037116 | AAV Gene Therapy for noise induced hearing loss using cerebrospinal fluid as route of delivery | , , , , , , , , , , , |
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Noise-induced hearing loss (NIHL) is one of the most prevalent disabilities for which effective therapeutic treatment is currently lacking. Auditory injury caused by excessive or constant noise exposure damages the sensory elements of the ear, leading to metabolic or mechanical damage to hair cells and subsequent degeneration of spiral ganglion neurons (SGNs). To improve hearing in the NIHL patient population, maintenance and regeneration of inner hair cell-SGN synapses is critical. Although effective compounds for the treatment of NIHL have been suggested, challenges in delivery of the compounds to the inner hair cells (IHCs) hinder translation of these therapies to a clinical setting. Current routes of administration are ineffective in reaching IHCs or are invasive and may damage the cochlea. In this study, we examined less invasive delivery routes, via intrathecal (cerebrospinal fluid delivery) or intravenous injection, to administer adeno-associated virus serotype 9 (AAV9) expressing green fluorescent protein (GFP) to target IHCs and SGNs. To induce auditory injury, mice were exposed to 100 dB sound pressure level (SPL) octave band noise for 2 hours. Mice were then injected at various time points post noise injury to determine if noise exposure influenced targeting efficiency. Three weeks post injection, cochleae were processed and analyzed for GFP expression using immunofluorescence. Our results demonstrate that intravenous delivery was not successful in targeting of IHCs or SGNs at any time point measured. However, intrathecally injected AAV9 was highly effective in targeting cells in both healthy and noise-damaged cochleae at multiple time points post-noise exposure. We conclude that gene therapy techniques can be utilized to efficiently deliver therapeutic transgenes to the cochlea using cerebrospinal fluid as a delivery route. These findings determine a novel route of delivery and open a new avenue of therapeutic treatment for auditory injury and cochlear disorders. |
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sciforum-037217 | Rapid reprogramming of ALS patient fibroblasts differentiates CuATSM responders from nonresponders. | , , , , , , , , , , | N/A |
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Patient diversity and unknown disease cause are major challenges for drug development and clinical trial design for ALS. Moreover, the heterogeneity of the ALS patient population is not reflected in currently available transgenic animal models making direct translation of potential therapeutics difficult. To address this, we utilize direct conversion technology to transform skin biopsies from ALS patients into neuronal progenitor cells (NPC). Using induced astrocytes (iAs) differentiated from these NPCs in co-culture with mouse embryonic motor neurons, we developed an in vitro ALS model to screen potential therapeutics. We have screened numerous compounds on multiple sporadic (sALS) and familial (fALS, mtSOD1 and C9ORF72) iAs and observed a diverse patient response to different therapeutic agents. Here, we utilize the compound, CuATSM, to subgroup therapeutic responsiveness of patient lines and identified shared dysfunctional pathways between responders. Following detailed analysis of ALS disease markers, elevated mitochondrial activity states was present in all CuATSM responders and was nonexistent in nonresponders. Treatment of iAs with CuATSM restored this activity to healthy control levels. Together, these findings suggest that iAs can be used to identify both disease modifiers and pathways dysregulated in an individual. Thus enhanced understanding of cellular profiles could facilitate personalized medicine based treatments. |
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sciforum-037963 | The role of paraoxonase 1 (PON1) as an oxidation marker, in muscle improvement after increased beta-hydroxybutyrate (BHB) in blood, in patients with multiple sclerosis | , |
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Various studies have concluded that in multiple sclerosis (MS) metabolic changes related to energy alterations are generated at the mitochondrial level. These changes produce loss of muscle mass and lead to high levels of oxidative stress related to low levels of the enzyme Paraoxonase 1 (PON1). The ketone bodies, mainly beta-hydroxybutyrate (BHB), restore this energetic alteration by improving muscular activity. This pilot study was conducted over 4 months with 51 patients diagnosed with MS, randomly divided into: an intervention group that received an isocaloric diet of a Mediterranean origin supplemented with 60 ml of coconut oil; and a control group that received the same base diet but without coconut oil. Before and after treatment, anthropometric measurements and blood samples were taken, measuring serum PON1 and BHB concentrations, to determine the role of PON1 in muscle improvement after increased blood BHB in MS patients. A significant increase in PON1 was obtained, associated with an increase in lean mass and an increase in BHB, so it was concluded that this enzyme appeared to be a good marker of decreased oxidation status in MS patients showing muscle improvement after increased BHB in blood. |
List of Authors (53)
Event Awards
Session | Title | Authors | Awards |
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Cognitive Neuroscience |
The role of paraoxonase 1 (PON1) as an oxidation marker, in muscle improvement after increased beta-hydroxybutyrate (BHB) in blood, in patients with multiple sclerosis | Best Paper Award | |
Molecular and Cellular Neuroscience |
Rapid reprogramming of ALS patient fibroblasts differentiates CuATSM responders from nonresponders. | Best Poster Award |
To acknowledge the support of the conference esteemed authors and recognize their outstanding scientific accomplishments, we are pleased to launch the Best Paper Award ,Best Poster Award and Best Speaker Award.
The Awards
Number of Awards Available: 1
Best paper award 500 CHF : The Best Paper Award is given for the paper judged to make the most significant contribution to the conference.Number of Awards Available: 1
Best poster award 500 CHF: The Best Poster Award has been established to recognize the scientific merit exhibited in poster presentation and preparation (For one candidates, with videos uploaded).Number of Awards Available: 1
Best speaker award 500 CHF: The Best Speaker Award is given for the speaker judged to make the most significant contribution to the conference.Terms and Conditions:
Best Paper Award
As a sponsor, Brain Sciences would like to award the best paper as elected by all the conference committee. The award will consist of 500 Swiss Francs. We look forward to posting your contributions.
Criteria for Evaluation of Best Paper Award:
Criteria
- Full paper must be submitted to IECBS 2020;
- Originality/Novelty of the paper;
- Significance of Content;
- Scientific Soundness;
- Interest to the readers;
- English language and style.
Evaluation
- Each Evaluation Committee member will give an assessment for each applicant in terms of the criteria outlined above;
- Total score for each presentation will be ranked, from highest to lowest;
- If two or more authors get the same score, further evaluation will be carried out;
- All decisions made by the Evaluation Committee are final.
Best Poster Award
As a sponsor, Brain Sciences would like to grants an award (500 Swiss Francs) for the best poster presented at the conference. This prize is awarded by a jury to the best designed poster presented at the conference.
Posters should have the following information.
- Title (with authors and affiliations)
- Introduction / Objectives / Aims
- Methods
- Results
- Conclusion
- References
- Acknowledgements
- Contact information
- 3-Minutes video presentation
During the conference, the chair is invited to judge the quality of the 3-minutes video presentation and poster. Criteria of judgement on the presentation shall be the ability of summarizing the content of the work and motivating the interest in looking at the poster. Criteria of judgement on the poster should be clarity of poster and appearance quality.
Best Speaker Award
The 1st International Electronic Conference on Brain Sciences (IECBS 2020) - Editor Interview video
Prof. Dr. Stephen D Meriney
3. Systems Neuroscience
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