Effective AAV-mediated gene replacement therapy in retinal organoids modeling AIPL1-associated LCA4

• Main Authors: Hali Sai (Author), Bethany Ollington (Author), Farah O. Rezek (Author), Niuzheng Chai (Author), Amelia Lane (Author), Anastasios Georgiadis (Author), James Bainbridge (Author), Michel Michaelides (Author), Almudena Sacristan-Reviriego (Author), Pedro R.L. Perdigão (Author), Amy Leung (Author), Jacqueline van der Spuy (Author)
• Format: Book
• Published: Elsevier, 2024-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Biallelic variations in the aryl hydrocarbon receptor interacting protein-like 1 (AIPL1) gene cause Leber congenital amaurosis subtype 4 (LCA4), an autosomal recessive early-onset severe retinal dystrophy that leads to the rapid degeneration of retinal photoreceptors and the severe impairment of sig...