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...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:N Engl J Med
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2017
Témata:
Article
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky