The United States of America, As Represented by the Secretary, Department of Health and Human Services

The Usa, As Represented by the Secretary, Dhhs

Suja Hiriyanna

Anand Swaroop

Zhijian Wu

Suddhasil Mookherjee

Tiansen Li

Disclosed are adeno-associated virus (AAV) vectors comprising a nucleotide sequence encoding RP2 or RPGR-ORF15 and related pharmaceutical compositions. Also disclosed are methods of treating or preventing X-linked retinitis pigmentosa, increasing photoreceptor number in a retina of a mammal, and increasing visual acuity of a mammal using the vectors and pharmaceutical compositions.

RP2 and RPGR vectors for treating X-linked retinitis pigmentosa

Expression vectors, viral particles and therapeutic methods of using such constructs to improve the visual function of a patient suffering from diseases of the eye, resulting from failure to produce a specific protein in the eye, or the production of a non-functional protein in the eye, particularly Leber Congenital Amaurosis (LCA) and CEP290-related LCA.

Methods and compositions for treating Leber congenital amaurosis

Methods and compositions for treating leber congenital amaurosis

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Boyds, MD, United States of America

Suja Hiriyanna