Patients with XLRP treated with AGTC-501 gene therapy experienced improvements in visual function including retinal sensitivity . . .

A systematic literature review was conducted to assess available data on disease progression in RPGR-associated XLRP.

Formulate recommendations for designing XLRP gene therapy clinical studies to provide the best chance of successful treatment development.

Among the most common genetic causes of IRDs are mutations in the RPGR gene located on the X-chromosome.

X-linked RP caused by RPGR mutations, an attractive target for gene therapy because of its clinical severity and large number of patients.

RPGR Gene Therapy Shows Statistically Significant & Continued Vision Improvement

Investigate X-linked retinal degeneration family with atypical preservation of visual acuity in the presence of a novel deep intronic splice

Zhimeng Zhang, Hehua Dai, Lei Wang, Tianchang Tao, Jing Xu, Xiaowei Sun, Liping Yang, Genlin Li | BMC Ophthalmology | Vol. 19, Article #...

The current study focused on the cilia-specific role for ARL2BP in photoreceptor cells.

Results extended the mutation spectrum of known RP genes in Han Chinese, making a contribution to RP gene diagnosis and the pathogenetic . .

Regulates ciliary localization of Joubert syndrome-associated protein INPP5E Kollu N. Rao, Wei Zhang, Linjing Li, Manisha Anand, and...

RPGR as a critical to cilia and suggest that trafficking of INPP5E to cilia depends upon the interaction of RPGR with PDE6B.

RPGR mutations were assessed in families with XLRP and a strategy for analyzing highly repetitive mutational hot spots in exon ORF15.