Periodic Reporting for period 1 - DADA2GT (Development of gene therapy and genome editing strategies to treat adenosine deaminase 2 deficiency)

Adenosine deaminase 2 deficiency (DADA2) is a rare inherited disorder caused by autosomal recessive mutations in the ADA2 gene. Clinical manifestations include early-onset lacunar strokes, vasculitis/vasculopathy, systemic inflammation, immunodeficiency, and hematologic...