Article
Correcting Autoinflammation in STING-Associated Vasculopathy with Onset in Infancy (SAVI) by Human Stem Cell Genome-Editing
2024-10-21
Abstract excerpt
<title>Abstract</title> <p>Type I interferonopathies comprise a large group of Mendelian autoinflammatory diseases that generally lack effective treatments. STING-associated Vasculitis with onset in Infancy (SAVI) is a severe autosomal dominant disease due to gain-of-functon(GOF) mutations in the gene STING1, encoding for the Stimulator of Interferon Response CGAMP Interactor 1 (STING1). SAVI leads to a severe mu...
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Identifiers and source
- Literature Corpus work
- 3224db89-2e54-54e7-a147-30366a4ae539
- DOI
- 10.21203/rs.3.rs-5090716/v1
