Massachusetts Eye and Ear Infirmary

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

Ucl Business Plc

Tiansen Li

Xiaoqing Liu

Robin Ali

Xinhua Shu

Michael A Sandberg

Basil Pawlyk

Alan Finlay Wright

Anand Swaroop

Suja Hiriyanna

Zhijian Wu

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

Methods for treating a human subject who has X-linked Retinitis Pigmentosa (XLRP) or another clinically-defined ophthalmological condition due to a loss-of-function mutation in the gene encoding the retinitis pigmentosa GTPase regulator (RPGR) protein, the method comprising administering to the subject a nucleic acid comprising an adeno-associated viral vector comprising an abbreviated human RPGR cDNA.

RPGR gene therapy for retinitis pigmentosa

Described are methods of treating and preventing conditions associated with a loss of elastic fibers. Also provided herein are methods of screening for agents useful in treating such conditions, and animal models of conditions associated with a loss of elastic fibers.

Lysyl oxidase-like 1 (LOXL1) and elastogenesis

Growing community of inventors

Quincy, MA, United States of America

Tiansen Li