CORDIS Project
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This project focuses on developing gene therapy for severe combined immunodeficiencies (SCIDs) using genome editing techniques. By employing CRISPR technology, the aim is to precisely correct genetic mutations in hematopoietic stem cells, paving the way for potential curative treatments for these life-threatening disor…
The severe combined immunodeficiencies (SCIDs) are a set of life threatening genetic diseases in which patients are born with mutations in single genes and are unable to develop functional immune systems.
While allogeneic bone marrow transplantation can be curative for these diseases, there remain significant limitations to this approach.
Gene therapy using viral vectors containing a corrective transgene is being developed for some of these disorders, most successfully for ADA-SCID.
However, for…
BAR ILAN UNIVERSITY
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