Gene/Cell Therapy R42 Active
Gene Therapy to Treat Ethanol-induced Osteoporosis Associated with Aldehyde Dehydrogenase 2 Deficiency in Post-menopausal East Asian Women CRYSTAL, RONALD · ENYX THERAPEUTICS, LLC · NEW YORK, NY
$1,253,597 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R01 Active
Nonhuman Primate Model of Inherited Photoreceptor Degeneration NEURINGER, MARTHA · OREGON HEALTH & SCIENCE UNIVERSITY · PORTLAND, OR
$767,366 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R01 Active
Development of a long-lasting, bone-targeted gene therapy for osteogenesis imperfecta Shim, Jae-Hyuck · UNIV OF MASSACHUSETTS MED SCH WORCESTER · WORCESTER, MA
$667,125 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R01 Active
Allele Specific Knockdown for LGMDD1 Findlay, Andrew · WASHINGTON UNIVERSITY · SAINT LOUIS, MO
$491,100 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R01 Active
Identifying regulatory uORFs as a targetable axis for hereditary disease Barash, Yoseph et al. · UNIVERSITY OF PENNSYLVANIA · PHILADELPHIA, PA
$404,023 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R01 Active
Intracerebroventricular cyclodextrin and AAV-mediated gene therapy forglobal CNS disease correction in feline NPC1 disease VITE, CHARLES · UNIVERSITY OF FLORIDA · GAINESVILLE, FL
$380,230 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R00 Active
In vivo precision genome editing to correct genetic disease Newby, Gregory · JOHNS HOPKINS UNIVERSITY · BALTIMORE, MD
$249,000 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R00 Active
First murine animal model and adeno-associated virus (AAV)-based gene therapy for MTATP6 mitochondrial diseases Ling, Qinglan · UNIV OF MASSACHUSETTS MED SCH WORCESTER · WORCESTER, MA
$245,217 (FY2025)
NIH RePORTER Show abstract Gene/Cell Therapy R21 Active
A one and done therapeutic strategy to correct ELP1 splicing defect in familial dysautonomia Alves, Christiano et al. · MASSACHUSETTS GENERAL HOSPITAL · BOSTON, MA
$203,290 (FY2026)
NIH RePORTER Show abstract Gene/Cell Therapy R21 Active
Anticodon Engineered tRNA for the Treatment of Hereditary Eye Disease Anderson, Michael · UNIVERSITY OF IOWA · IOWA CITY, IA
$187,260 (FY2026)
NIH RePORTER Show abstract 