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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Publicado en:Hum Gene Ther
Autores principales: 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
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc., publishers 2020
Materias:
Research Articles
Acceso en línea: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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