BioCentury | Aug 28, 2020
Distillery Therapeutics
CRISPR-based gene editing for Wiskott-Aldrich syndrome
DISEASE CATEGORY: Autoimmune INDICATION: Autoimmune Gene editing of mutated WASp in hematopoietic stem and progenitor cells (HSPCs) could treat Wiskott-Aldrich syndrome. The therapy uses an adeno-associated virus serotype 6 (AAV6) vector to deliver a CRISPR-Cas9...