Загрузка...
Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery
Mucopolysaccharidosis (MPS) IIIB is a lysosomal storage disease with complex CNS and somatic pathology due to a deficiency in α-N-acetylglucosaminidase (NAGLU). Using global metabolic profiling by mass spectrometry targeting 361 metabolites, this study detected significant decreases in 225 and incre...
Сохранить в:
| Опубликовано в: : | Mol Ther |
|---|---|
| Главные авторы: | , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
American Society of Gene & Cell Therapy
2017
|
| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5363204/ https://ncbi.nlm.nih.gov/pubmed/28143737 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2016.12.025 |
| Метки: |
Добавить метку
Нет меток, Требуется 1-ая метка записи!
|