Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

BACKGROUND: In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allo...

Fuld beskrivelse

Na minha lista:
Bibliografiske detaljer
Udgivet i:N Engl J Med
Main Authors: Eichler, Florian, Duncan, Christine, Musolino, Patricia L., Orchard, Paul J., De Oliveira, Satiro, Thrasher, Adrian J., Armant, Myriam, Dansereau, Colleen, Lund, Troy C., Miller, Weston P., Raymond, Gerald V., Sankar, Raman, Shah, Ami J., Sevin, Caroline, Gaspar, H. Bobby, Gissen, Paul, Amartino, Hernan, Bratkovic, Drago, Smith, Nicholas J.C., Paker, Asif M., Shamir, Esther, O’Meara, Tara, Davidson, David, Aubourg, Patrick, Williams, David A.
Format: Artigo
Sprog:Inglês
Udgivet: 2017
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5708849/
https://ncbi.nlm.nih.gov/pubmed/28976817
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1700554
Tags: Tilføj Tag
Ingen Tags, Vær først til at tagge denne postø!

Lignende værker