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...

Полное описание

Сохранить в:
Библиографические подробности
Опубликовано в: :Mol Ther
Главные авторы: 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.
Формат: Artigo
Язык:Inglês
Опубликовано: American Society of Gene & Cell Therapy 2019
Предметы:
Original Article
Online-ссылка: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
Метки: Добавить метку
Нет меток, Требуется 1-ая метка записи!

Схожие документы