MAGICNCT07889947
Combining Gene Editing and iPS to Study and Treat Monogenic Autoinflammatory Diseases
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Record History
All 1 version: 1 with changes below.
- Study First Submitted
Endpoints
Primary
To generate a model of type I interferonopathy to gain insight in STING1 role in nonhematopoietic cells and to explore gene editing as a therapeutic option
Timeframe: through study completion, an average of 2 years
Secondary (2)
To generate an isogenic iPSC line-based in vitro model for the V155M SAVI mutation using CRISPR/Cas9 GE and somatic cell differentiation to study organ-specific pathogenic
To develop a GE therapy approach using CRISPR/Cas9 base editing and CRISPR/Cas9 to obtain in locus gene correction of STING1 mutation in HSCs (Aim 2)
Arms and interventions
A patient with a genetically confirmed diagnosis of STING-associated vasculopathy
case controls
Sites
2 sites in 1 country