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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:N Engl J Med
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: 2017
Aiheet:
Article
Linkit: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
Tagit: Lisää tagi
Ei tageja, Lisää ensimmäinen tagi!

Samankaltaisia teoksia