Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Titel:
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Auteur:
Michaelides, Michel Laich, Yannik Wong, Sui Chien Oluonye, Ngozi Zaman, Serena Kumaran, Neruban Kalitzeos, Angelos Petrushkin, Harry Georgiou, Michalis Tailor, Vijay Pabst, Marc Staeubli, Kim Maimon-Mor, Roni O Jones, Peter R Scholte, Steven H Georgiadis, Anastasios van der Spuy, Jacqueline Naylor, Stuart Forbes, Alexandria Dekker, Tessa M Arulmuthu, Eugene R Smith, Alexander J Ali, Robin R Bainbridge, James W B
Verschenen in:
Lancet
Paginering:
Jaargang 405 () nr. 10479 pagina's 648-657
Jaar:
2025
Inhoud:
Uitgever:
The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY 4.0 license