X-Linked Retinitis Pigmentosa

X-linked retinitis pigmentosa (XLRP) is inherited condition that causes progressive vision loss in boys and young men.  The condition begins with night blindness in young boys followed by progressive constriction of the field of vision.  Affected men become legally blind at an average of about 45 years of age.

The most common form of XLRP is caused by mutations in the RPGR gene, and AGTC is developing a gene therapy product for this form of XLRP.  Treatment with a gene therapy product slowed the loss of visual function in dogs with XLRP caused by mutations in the RPGR gene.  Based on the strong preclinical proof-of-concept data, AGTC has initiated a formal preclinical testing program that will lead to clinical evaluation of this product.

Related Publications

Evaluation of AAV2tYF-GRK1-RPGR Vectors in a Canine Model of RPGR-XLRP

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. 

RPGR-associated retinal degeneration in human X-linked RP and a murine model. 

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