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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Bibliographic Details
Main Authors:	Lim, WF, Forouhan, M, Roberts, TC, Dabney, J, Ellerington, R, Speciale, AA, Manzano, R, Lieto, M, Sangha, G, Banerjee, S, Conceição, M, Cravo, L, Biscans, A, Roux, L, Pourshafie, N, Grunseich, C, Duguez, S, Khvorova, A, Pennuto, M, Cortes, CJ, La Spada, AR, Fischbeck, KH, Wood, MJA, Rinaldi, C
Format:	Journal article
Language:	English
Published:	American Association for the Advancement of Science 2021

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

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