iPSC-Derived LCHADD Retinal Pigment Epithelial Cells Are Susceptible to Lipid Peroxidation and Rescued by Transfection of a Wildtype AAV-<i>HADHA</i> Vector
iPSC-derived LCHADD-RPE are susceptible to lipid peroxidation mediated cell death and are rescued by exogenous HADHA delivered with rAAV. These results are promising for AAV-HADHA gene addition therapy as a possible treatment for chorioretinopathy in patients with LCHADD.
Authors
- Melanie B. Gillingham (ORCID: https://orcid.org/0000-0003-0897-8268)
- Cary O. Harding (ORCID: https://orcid.org/0000-0001-9146-3112)
- Mikhail S. Shchepinov (ORCID: https://orcid.org/0000-0001-6154-1212)
- Shannon Babcock (ORCID: https://orcid.org/0000-0003-2365-2119)
- Tiffany DeVine
- Dietrich Matern (ORCID: https://orcid.org/0000-0001-6298-4138)
- Gabriela Elizondo
- Mark E. Pennesi (ORCID: https://orcid.org/0000-0002-4943-6599)
- Garen Gaston
Institutions
- École Nationale Supérieure de Chimie de Montpellier (FR)
- Skolkovo Institute of Science and Technology (RU)
- Centre National de la Recherche Scientifique (FR)
- Mayo Clinic (US)
- Oregon Health & Science University (US)
- Université de Montpellier (FR)
- Retina Foundation of the Southwest (US)
- Institut des Biomolécules Max Mousseron (FR)
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2024-09-16
- DOI
- https://doi.org/10.1167/iovs.65.11.22
- Citations
- 3
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.70
Funders
- National Institutes of Health