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Small molecule splicing modifiers with systemic HTT-lowering activity.
Bhattacharyya, Anuradha; Trotta, Christopher R; Narasimhan, Jana; Wiedinger, Kari J; Li, Wencheng; Effenberger, Kerstin A; Woll, Matthew G; Jani, Minakshi B; Risher, Nicole; Yeh, Shirley; Cheng, Yaofeng; Sydorenko, Nadiya; Moon, Young-Choon; Karp, Gary M; Weetall, Marla; Dakka, Amal; Gabbeta, Vijayalakshmi; Naryshkin, Nikolai A; Graci, Jason D; Tripodi, Thomas; Southwell, Amber; Hayden, Michael; Colacino, Joseph M; Peltz, Stuart W.
Afiliação
  • Bhattacharyya A; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Trotta CR; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Narasimhan J; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Wiedinger KJ; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Li W; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Effenberger KA; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Woll MG; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Jani MB; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Risher N; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Yeh S; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Cheng Y; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Sydorenko N; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Moon YC; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Karp GM; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Weetall M; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Dakka A; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Gabbeta V; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Naryshkin NA; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Graci JD; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Tripodi T; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Southwell A; Burnett School of Biomedical Sciences, College of Medicine, University of Central Florida, Orlando, FL, USA.
  • Hayden M; Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada.
  • Colacino JM; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA.
  • Peltz SW; PTC Therapeutics, Inc. 100 Corporate Court, South Plainfield, NJ, USA. speltz@ptcbio.com.
Nat Commun ; 12(1): 7299, 2021 12 15.
Article em En | MEDLINE | ID: mdl-34911927

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Splicing de RNA / Doença de Huntington / Bibliotecas de Moléculas Pequenas / Proteína Huntingtina Limite: Animals / Humans Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Splicing de RNA / Doença de Huntington / Bibliotecas de Moléculas Pequenas / Proteína Huntingtina Limite: Animals / Humans Idioma: En Revista: Nat Commun Assunto da revista: BIOLOGIA / CIENCIA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos