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Ophthalmology|June 17, 2022
Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy TrialTomas S Aleman, Rachel M Huckfeldt, Leona W Serrano, et al.
Human Gene Therapy|December 7, 2011
Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of conceptAstra Dinculescu, Jackie Estreicher, Juan C Zenteno, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 29, 2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injectionSamuel G Jacobson, Gregory M Acland, Gustavo D Aguirre, et al.
Investigative Ophthalmology & Visual Science|December 23, 2021
Comparative Natural History of Visual Function From Patients With Biallelic Variants in BBS1 and BBS10Monika K Grudzinska Pechhacker, Samuel G Jacobson, Arlene V Drack, et al.
Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|January 13, 2022
BAMM (BRAF Autophagy and MEK Inhibition in Melanoma): A Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine in Advanced BRAFV600-mutant MelanomaJanice M Mehnert, Tara C Mitchell, Alexander C Huang, et al.
Archives of Ophthalmology (Chicago, Ill. : 1960)|September 14, 2011
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 yearsSamuel G Jacobson, Artur V Cideciyan, Ramakrishna Ratnakaram, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 2, 2025
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trialTomas S Aleman, Katherine E Uyhazi, Alejandro J Roman, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 21, 2018
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital BlindnessJi Yun Song, Puya Aravand, Sergei Nikonov, et al.
Investigative Ophthalmology & Visual Science|December 18, 2024
Retinal Degeneration Associated With Biallelic RDH12 Variants: Longitudinal Evaluation of Retinal Structure and Visual Function in Pediatric PatientsTomas S Aleman, Alejandro J Roman, Katherine E Uyhazi, et al.
Plos Genetics|May 4, 2019
Variants in myelin regulatory factor (MYRF) cause autosomal dominant and syndromic nanophthalmos in humans and retinal degeneration in miceSarah J Garnai, Michelle L Brinkmeier, Ben Emery, et al.
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