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

Author:

Lim Wooi F.1ORCID,Forouhan Mitra1,Roberts Thomas C.1ORCID,Dabney Jesse2ORCID,Ellerington Ruth1,Speciale Alfina A.1ORCID,Manzano Raquel2ORCID,Lieto Maria2,Sangha Gavinda2ORCID,Banerjee Subhashis2,Conceição MarianaORCID,Cravo Lara1ORCID,Biscans Annabelle3ORCID,Roux Loïc2ORCID,Pourshafie Naemeh4,Grunseich Christopher4,Duguez Stephanie5ORCID,Khvorova Anastasia3,Pennuto Maria67ORCID,Cortes Constanza J.8,La Spada Albert R.9ORCID,Fischbeck Kenneth H.4ORCID,Wood Matthew J.A.110ORCID,Rinaldi Carlo110ORCID

Affiliation:

1. Department of Paediatrics, University of Oxford, Oxford, UK.

2. Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK.

3. RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.

4. Neurogenetics Branch, National Institute of Neurological Disorders and Stroke (NINDS), Bethesda, MD, USA.

5. Northern Ireland Centre for Stratified Medicine, Biomedical Sciences Research Institute, Londonderry, UK.

6. Department of Biomedical Sciences, University of Padova, Padova, Italy.

7. Venetian Institute of Molecular Medicine (VIMM), Padova, Italy.

8. Department of Neurology, Duke Center for Neurodegeneration and Neurotherapeutics, Duke University School of Medicine, Durham, NC, USA.

9. Departments of Pathology and Laboratory Medicine, Neurology, and Biological Chemistry and the UCI Institute for Neurotherapeutics, University of California, Irvine, Irvine, CA, USA.

10. MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, UK.

Abstract

AR isoform 2–encoded variant acts as a transcriptional modulator of AR genomic activity and is a target for therapy for SBMA.

Funder

Wellcome

Medical Research Council Canada

MDA

AFM

Telethon

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3