AAV-mediated gene replacement therapy for LRAT-associated retinitis pigmentosa: a proof-of-concept study in a patient-based rat model

Gene Therapy 2026

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Authors: A. M. El-Kalaani, Jacoline B. ten Brink, Camiel J. F. Boon, Céline Koster

DOI: 10.1038/s41434-026-00601-9

Cited by: 0

References: 68

Fields of Study & Subjects

Retinal Development and Disorders Virus-based gene therapy research RNA Interference and Gene Delivery

References 68