Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy

Globoid cell leukodystrophy (GLD) is a demyelinating lysosomal storage disease due to the deficiency of the galactocerebrosidase (GALC) enzyme. The favorable outcome of hematopoietic stem and progenitor cell (HSPC)-based approaches in GLD and other similar diseases suggests HSPC gene therapy as a pr...

Полное описание

Сохранить в:
Библиографические подробности
Опубликовано в: :Mol Ther Methods Clin Dev
Главные авторы: Ungari, Silvia, Montepeloso, Annita, Morena, Francesco, Cocchiarella, Fabienne, Recchia, Alessandra, Martino, Sabata, Gentner, Bernhard, Naldini, Luigi, Biffi, Alessandra
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2015
Предметы:
Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4605225/
https://ncbi.nlm.nih.gov/pubmed/26509184
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.38
Метки: Добавить метку
Нет меток, Требуется 1-ая метка записи!

Схожие документы