Combined Expression of hRdCVF and hRdCVFL Through AAV-Mediated Delivery for the Treatment of Retinitis Pigmentosa
This study revealed that both proteins encoded by NXNL1 are essential for gene-independent RP treatment and that combining RdCVF and RdCVFL delivery with a single AAV vector yields a more substantial photoreceptor-protective effect. This functional proof of concept supports the ongoing gene therapy phase 1/2 trial Promising ROd-cone DYstrophy Gene therapY (PRODYGY, NCT05748873).
Authors
- Imen Harichane
- Seiki Achiedo
- C Zeitz
- JS Bennett
- Delphine Pagan (ORCID: https://orcid.org/0000-0002-8298-3682)
- Ying Yang (ORCID: https://orcid.org/0000-0002-9137-4889)
- Quénol Cesar
- Manuel Simonutti
- Frederic Blond
- Julie Dégardin
- Thérèse Cronin
- Isabelle Audo
- Thierry Léveillard
- Géraldine Millet-Puel
- Najate Aït-Ali
- José-Alain Sahel
Institutions
- Centre National de la Recherche Scientifique (FR)
- Institut de la Vision (FR)
- Inserm (FR)
- University of Pittsburgh (US)
- Sorbonne Université (FR)
- Laboratoire National de Référence (MA)
- Translational Therapeutics (United States) (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2026-03-02
- DOI
- https://doi.org/10.1167/iovs.67.3.2
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- University of Pennsylvania
- Institut National de la Santé et de la Recherche Médicale
- Sorbonne Université
- Inserm Transfert