Mark Basche

A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency

Julie Tordo, Claire O’Leary, André Saraiva Leão Marcelo Antunes et al. · Brain · 2018 · 123 citations · Full text

Virus Structure, Engineering, Molecular Virology +14

Restoration of visual function in advanced disease after transplantation of purified human pluripotent stem cell-derived cone photoreceptors

Joana Ribeiro, Christopher A. Procyk, Emma L. West et al. · Cell Reports · 2021 · 120 citations · Full text

Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

Mei Hong Tan, Alexander J. Smith, Basil S. Pawlyk et al. · Human Molecular Genetics · 2009 · 117 citations · Full text

Nanotube‐like processes facilitate material transfer between photoreceptors

Aikaterini A. Kalargyrou, Mark Basche, Aura Hare et al. · EMBO Reports · 2021 · 88 citations · Full text

Successful Gene Therapy in Older Rpe65-Deficient Dogs Following Subretinal Injection of an Adeno-Associated Vector Expressing <i>RPE65</i>

Matthew J. Annear, Freya M. Mowat, Joshua T. Bartoe et al. · Human Gene Therapy · 2013 · 33 citations

Ocular Disease, In Vivo Gene Therapy, Immunology +16