Suk See De Ravin

The paper identifies a novel PIK3R1 frameshift mutation linked to immunodeficiency and ALS, showing haploinsufficiency causes neuronal dysfunction and apoptosis.

This study compares CRISPR/HDR and lentiviral transduction effects on HSPC engraftment and clonal dynamics in rhesus macaques, finding HDR-edited cells decline rapidly while lentiviral cells persist longer.

This paper reports a case of clonal hematopoiesis in a rhesus macaque following lentiviral transduction of HSPC with a strong retroviral promoter, leading to gene overexpression and aberrant splicing.