May 24, 2017Molecular TherapyOpen Access

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

View Full Paper

Ask AI

Bookmark

Abstract

) and shows good safety in C57BL6/J wild-type mice. This work provides the basis for clinical trial development to treat patients with XLRP caused by RPGR mutations.

Discussion

No takes yet. Share an insight, caveat, or question.

Cite This Study

Fischer et al. (2017) studied this question.

synapsesocial.com/papers/69ffd2d464548b97a42d73f0 https://doi.org/10.1016/j.ymthe.2017.05.005

View Full Paper

Ask AI

Bookmark

Abstract

) and shows good safety in C57BL6/J wild-type mice. This work provides the basis for clinical trial development to treat patients with XLRP caused by RPGR mutations.

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

Abstract

Discussion

Cite This Study

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

Abstract

Discussion

Cite This Study

Keep reading

Keep reading