Gene therapy for leber congenital amaurosis caused by RPE65 mutations: Safety and efficacy in 15 children and adults followed up to 3 years

Samuel G. Jacobson, Artur V. Cideciyan, Ramakrishna Ratnakaram, Elise Heon, Sharon B. Schwartz, Alejandro J. Roman, Marc C. Peden, Tomas S. Aleman, Sanford L. Boye, Alexander Sumaroka, Thomas J. Conlon, Roberto Calcedo, Ji Jing Pang, Kirsten E. Erger, Melani B. Olivares, Cristina L. Mullins, Malgorzata Swider, Shalesh Kaushal, William J. Feuer, Alessandro IannacconeGerald A. Fishman, Edwin M. Stone, Barry J. Byrne, William W. Hauswirth

Research output: Contribution to journalArticlepeer-review

455 Scopus citations

Fingerprint

Dive into the research topics of 'Gene therapy for leber congenital amaurosis caused by RPE65 mutations: Safety and efficacy in 15 children and adults followed up to 3 years'. Together they form a unique fingerprint.

Medicine & Life Sciences