Prevention of Photoreceptor Cell Loss in a Cln6(nclf) Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells

The neuronal ceroid lipofuscinoses (NCLs) are inherited lysosomal storage disorders characterized by general neurodegeneration and premature death. Sight loss is also a major symptom in NCLs, severely affecting the quality of life of patients, but it is not targeted effectively by brain-directed the...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: kleine Holthaus, Sophia-Martha, Ribeiro, Joana, Abelleira-Hervas, Laura, Pearson, Rachael A., Duran, Yanai, Georgiadis, Anastasios, Sampson, Robert D., Rizzi, Matteo, Hoke, Justin, Maswood, Ryea, Azam, Selina, Luhmann, Ulrich F.O., Smith, Alexander J., Mole, Sara E., Ali, Robin R.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5993939/
https://ncbi.nlm.nih.gov/pubmed/29606505
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.02.027
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