Clinical Trial: Combining Gene Editing and iPS to Study and Treat Monogenic Autoinflammatory Diseases

U.S., Oct. 23 -- ClinicalTrials.gov registry received information related to the study (NCT07889947) titled 'Combining Gene Editing and iPS to Study and Treat Monogenic Autoinflammatory Diseases' on Oct. 12.

Brief Summary: Type I interferonopathies (T1I) are rare autoinflammatory diseases characterized by dysregulated type I interferon signaling, leading to severe multisystem inflammation, including skin and lung involvement. STING-associated vasculopathy with onset in infancy (SAVI) is an autosomal dominant T1I associated with severe systemic inflammation, skin ulcers, and progressive interstitial lung disease. Current treatments are often insufficient, highlighting the need for novel therapeutic strategies. This project aims to develop p...