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Dog models for blinding inherited retinal dystrophies.


ABSTRACT: Spontaneous canine models exist for several inherited retinal dystrophies. This review will summarize the models and indicate where they have been used in translational gene therapy trials. The RPE65 gene therapy trials to treat childhood blindness are a good example of how studies in dogs have contributed to therapy development. Outcomes in human clinical trials are compared and contrasted with the result of the preclinical dog trials.

SUBMITTER: Petersen-Jones SM 

PROVIDER: S-EPMC4442585 | biostudies-literature | 2015 Mar

REPOSITORIES: biostudies-literature

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Dog models for blinding inherited retinal dystrophies.

Petersen-Jones Simon M SM   Komáromy András M AM  

Human gene therapy. Clinical development 20150211 1


Spontaneous canine models exist for several inherited retinal dystrophies. This review will summarize the models and indicate where they have been used in translational gene therapy trials. The RPE65 gene therapy trials to treat childhood blindness are a good example of how studies in dogs have contributed to therapy development. Outcomes in human clinical trials are compared and contrasted with the result of the preclinical dog trials. ...[more]

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