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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Pubblicato in:Mol Ther
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2018
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Original Article
Accesso online: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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