Long-Term Effects of Gene Therapy in a Novel Mouse Model of Human MFRP-Associated Retinopathy

Patients harboring homozygous c.498_499insC mutations in MFRP demonstrate hyperopia, microphthalmia, retinitis pigmentosa, retinal pigment epithelial atrophy, variable degrees of foveal edema, and optic disc drusen. The disease phenotype is variable, however, with some patients maintaining good cent...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Chekuri, Anil, Sahu, Bhubanananda, Chavali, Venkata Ramana Murthy, Voronchikhina, Marina, Soto-Hermida, Angel, Suk, John J., Alapati, Akhila N., Bartsch, Dirk-Uwe, Ayala-Ramirez, Raul, Zenteno, Juan C., Dinculescu, Astra, Jablonski, Monica M., Borooah, Shyamanga, Ayyagari, Radha
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2019
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6534092/
https://ncbi.nlm.nih.gov/pubmed/30499344
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2018.192
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