AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity

Adeno-associated virus-mediated gene replacement is emerging as a safe and effective means of correcting single-gene mutations affecting the CNS. AAV-mediated progranulin gene (GRN) delivery has been proposed as a treatment for GRN-deficient frontotemporal dementia and neuronal ceroid lipofuscinosis...

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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: Amado, Defne A., Rieders, Julianne M., Diatta, Fortunay, Hernandez-Con, Pilar, Singer, Adina, Mak, Jordan T., Zhang, Junxian, Lancaster, Eric, Davidson, Beverly L., Chen-Plotkin, Alice S.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2019
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6369714/
https://ncbi.nlm.nih.gov/pubmed/30559071
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.11.013
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