Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease

The neuronal ceroid lipofuscinoses (NCLs), often referred to as Batten disease, are inherited lysosomal storage disorders that represent the most common neurodegeneration during childhood. Symptoms include seizures, vision loss, motor and cognitive decline, and premature death. The development of br...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther
Main Authors: kleine Holthaus, Sophia-Martha, Aristorena, Mikel, Maswood, Ryea, Semenyuk, Olha, Hoke, Justin, Hare, Aura, Smith, Alexander J., Mole, Sara E., Ali, Robin R.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc., publishers 2020
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Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7404834/
https://ncbi.nlm.nih.gov/pubmed/32578444
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2020.038
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