Nalaganje...

Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus–α-L-Iduronidase for Hurler Disease

BACKGROUND: Hurler disease (mucopolysaccharidosis type I [MPS-I]) is an inherited metabolic disorder characterized by deficiency of the lysosomal enzyme α-L-iduronidase (IDUA). Currently, the only therapies for MPS-I, enzyme replacement and hematopoietic stem cell transplantation, are generally inef...

Popoln opis

Shranjeno v:
Bibliografske podrobnosti
Main Authors: Janson, Christopher G., Romanova, Liudmila G., Leone, Paola, Nan, Zhenhong, Belur, Lalitha, McIvor, R. Scott, Low, Walter C.
Format: Artigo
Jezik:Inglês
Izdano: 2014
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4116107/
https://ncbi.nlm.nih.gov/pubmed/24077583
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1227/NEU.0000000000000157
Oznake: Označite
Brez oznak, prvi označite!