Gene Therapy Improves Functional Measures in XLRP
Patients with XLRP treated with AGTC-501 gene therapy experienced improvements in visual function including retinal sensitivity . . .
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- Jan 31
A Systematic Literature Review of Disease Progression Reported in RPGR-associated X-Linked Retinitis Pigmentosa
A systematic literature review was conducted to assess available data on disease progression in RPGR-associated XLRP.
- Jun 9, 2023
Overcoming the Challenges to Clinical Development of X-Linked Retinitis Pigmentosa Therapies: Proceedings of an Expert Panel
Formulate recommendations for designing XLRP gene therapy clinical studies to provide the best chance of successful treatment development.
- Apr 3, 2003
X-linked Retinitis Pigmentosa: RPGR Mutations in Most Families with Definite X Linkage and Clustering of Mutations in a Short Sequence Stretch of Exon ORF15
RPGR mutations were assessed in families with XLRP and a strategy for analyzing highly repetitive mutational hot spots in exon ORF15.
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