Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity

Spinal and bulbar muscular atrophy (SBMA) is an X-linked, adult-onset neuromuscular condition caused by an abnormal polyglutamine (polyQ) tract expansion in androgen receptor (AR) protein. SBMA is a disease with high unmet clinical need. Recent studies have shown that mutant AR-altered transcription...

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Detalles Bibliográficos
Autores principales: Lim, Wooi F., Forouhan, Mitra, Roberts, Thomas C., Dabney, Jesse, Ellerington, Ruth, Speciale, Alfina A., Manzano, Raquel, Lieto, Maria, Sangha, Gavinda, Banerjee, Subhashis, Conceição, Mariana, Cravo, Lara, Biscans, Annabelle, Roux, Loïc, Pourshafie, Naemeh, Grunseich, Christopher, Duguez, Stephanie, Khvorova, Anastasia, Pennuto, Maria, Cortes, Constanza J., La Spada, Albert R., Fischbeck, Kenneth H., Wood, Matthew J.A., Rinaldi, Carlo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8378820/
https://www.ncbi.nlm.nih.gov/pubmed/34417184
http://dx.doi.org/10.1126/sciadv.abi6896

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