Transfection strategy for Haematopoietic Stem cells to treat rare blood diseases like Sickle Cell Anaemia: Aratrika Halder^a_, department of Oils Lipids Science and technology, CSIR-IICT, Hyderabad-500076

Sickle Cell Anaemia is a rare blood disease where mutation occurs in the beta-globin chain of chromosome number 11. As a result glutamic acid is replaced by valine and, hence, the RBCs appear sickle-shaped. Sialic acids are very much overexpressed on sickle cell RBCs and are an easy therapeutic target.

This study demonstrates the synthesis of such a sialic-acid lipid-targeting ligand and its formulation with other transfecting agents to strategically transfect Haematopoietic Stem cells via egfp for developing a strategy to curb Sickle Cell Disease. The LNP formulations were checked for particle size and charge through a DLS experiment. The molecules synthesised were characterised using NMR spectroscopy and mass spectroscopy.

In this study we isolated Haematopoietic Stem cells from mice bone marrow and treated them with different formulations to check the transfection qualitatively and quantitatively. Flow Cytometric Analysis and Confocal Microscopy were performed to co-relate the data.

We concluded the study by confirming an adequate amount of Haematopoietic Stem cells were transfected through our formulation and, hence, these formulations can be an effective strategy to transfect Haematopoietic Stem cells of Sickle Cell Anaemia patients.

This study has a future scope for experiments in Human Haematopoietic Stem cells and could lead to clinical trials for developing Sickle Cell Anaemia vaccines.