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CRISPR-Cas9–based gene editing as a proof-of-concept approach in an inborn error of immunity caused by a DCLRE1C variantNARA Subscribed
Hypomorphic DCLRE1C variants impair T and B cell development, leading to combined immunodeficiency (CID) or leaky severe combined immunodeficiency (SCID). Current treatment options, such as allogeneic hematopoietic stem cell transplantation (aHSCT), are associated with significant risks, highlighting the need for alternative therapeutic strategies. In this study, we report the first a proof-of-concept CRISPR-Cas9–mediated corr...
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