Gene editing using CRISPR nucleases
Monogenic inherited disorders can be treated ex vivo by correction or inactivation of the mutated gene using CRISPR nucleases mediated gene editing in autologous hematopoietic stem cells (HSCs), followed by the transplantation of the gene-corrected HSCs back into the patient without exposure to harsh immunosuppression regimes. Besides the treatment of monogenic diseases, gene editing may be applied as a new therapy for common disease in the fields of oncology, neurology, neurosensory, autoimmune diseases, viral infections, and also as a novel diagnostic tool.
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