Development of a Gene Therapy Vector for RDH12-Associated Retinal Dystrophy

Early-onset severe retinal dystrophy (EOSRD) is a genetically heterogeneous group of diseases resulting in serious visual disability in children. A significant number of EOSRD cases, often diagnosed as Leber congenital amaurosis (LCA13), are associated with mutations in the gene encoding retinol deh...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: Feathers, Kecia L., Jia, Lin, Perera, Nirosha Dayanthi, Chen, Adrienne, Presswalla, Feriel K., Khan, Naheed W., Fahim, Abigail T., Smith, Alexander J., Ali, Robin R., Thompson, Debra A.
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/PMC6854515/
https://ncbi.nlm.nih.gov/pubmed/31237438
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.017
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