Published in
American Association for the Advancement of Science, Science Advances, 34(7), 2021
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Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity
Journal article published in 2021 by
Wooi F. Lim 
,
Mitra Forouhan,
Thomas C. Roberts ,
Jesse Dabney ,
Ruth Ellerington,
Alfina A. Speciale ,
Raquel Manzano,
Maria Lieto,
Gavinda Sangha ,
Subhashis Banerjee,
Mariana Conceição ,
Lara Cravo ,
Annabelle Biscans ,
Loïc Roux ,
Naemeh Pourshafie
and other authors.
,
Mitra Forouhan,
Thomas C. Roberts ,
Jesse Dabney ,
Ruth Ellerington,
Alfina A. Speciale ,
Raquel Manzano,
Maria Lieto,
Gavinda Sangha ,
Subhashis Banerjee,
Mariana Conceição ,
Lara Cravo ,
Annabelle Biscans ,
Loïc Roux ,
Naemeh Pourshafie
and other authors.
This paper is made freely available by the publisher.
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Abstract
AR isoform 2–encoded variant acts as a transcriptional modulator of AR genomic activity and is a target for therapy for SBMA.