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Gene therapy for disorders of the extracellular matrix — Douglas Gould (UNIVERSITY OF CALIFORNIA, SAN FRANCISCO)

PROJECT SUMMARY COL4A1 and COL4A2 mutations cause Gould syndrome (GS) – a multisystem disorder for which clinically heterogeneous cerebrovascular disease is the major consequence. Cerebrovascular dise

Project titleGene therapy for disorders of the extracellular matrix
Project number1RF1NS128217-01A1
PI nameDouglas Gould
InstitutionUNIVERSITY OF CALIFORNIA, SAN FRANCISCO
Funding mechanismRF1
AgencyNINDS
Award amount$2.48M
Fiscal year2023
Project start2023-04-01T00:00:00
Project end2026-03-31T00:00:00
AbstractPROJECT SUMMARY COL4A1 and COL4A2 mutations cause Gould syndrome (GS) – a multisystem disorder for which clinically heterogeneous cerebrovascular disease is the major consequence. Cerebrovascular dise
Key terms<AAV vector><Ablation><Address><Affect><Alleles><Allelomorphs><Aminoacetic Acid><Antimorphic mutatio

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