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Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.
Zeitler, Bryan; Froelich, Steven; Marlen, Kimberly; Shivak, David A; Yu, Qi; Li, Davis; Pearl, Jocelynn R; Miller, Jeffrey C; Zhang, Lei; Paschon, David E; Hinkley, Sarah J; Ankoudinova, Irina; Lam, Stephen; Guschin, Dmitry; Kopan, Lexi; Cherone, Jennifer M; Nguyen, Hoang-Oanh B; Qiao, Guijuan; Ataei, Yasaman; Mendel, Matthew C; Amora, Rainier; Surosky, Richard; Laganiere, Josee; Vu, B Joseph; Narayanan, Anand; Sedaghat, Yalda; Tillack, Karsten; Thiede, Christina; Gärtner, Annette; Kwak, Seung; Bard, Jonathan; Mrzljak, Ladislav; Park, Larry; Heikkinen, Taneli; Lehtimäki, Kimmo K; Svedberg, Marie M; Häggkvist, Jenny; Tari, Lenke; Tóth, Miklós; Varrone, Andrea; Halldin, Christer; Kudwa, Andrea E; Ramboz, Sylvie; Day, Michelle; Kondapalli, Jyothisri; Surmeier, D James; Urnov, Fyodor D; Gregory, Philip D; Rebar, Edward J; Muñoz-Sanjuán, Ignacio.
Afiliação
  • Zeitler B; Sangamo Therapeutics, Inc., Richmond, CA, USA. bzeitler@sangamo.com.
  • Froelich S; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Marlen K; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Shivak DA; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Yu Q; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Li D; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Pearl JR; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Miller JC; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Zhang L; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Paschon DE; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Hinkley SJ; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Ankoudinova I; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Lam S; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Guschin D; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Kopan L; Laboratory of Intracellular Signalling, Moscow Institute of Physics and Technology, Dolgoprudnyi, Russian Federation.
  • Cherone JM; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Nguyen HB; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Qiao G; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Ataei Y; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Mendel MC; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Amora R; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Surosky R; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Laganiere J; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Vu BJ; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Narayanan A; Medical Affairs and Innovation, Hema-Quebec, Quebec City, Quebec, Canada.
  • Sedaghat Y; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Tillack K; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Thiede C; Evotec AG, Hamburg, Germany.
  • Gärtner A; Evotec AG, Hamburg, Germany.
  • Kwak S; Evotec AG, Hamburg, Germany.
  • Bard J; Evotec AG, Hamburg, Germany.
  • Mrzljak L; CHDI Management/CHDI Foundation, Los Angeles, CA, USA.
  • Park L; CHDI Management/CHDI Foundation, Los Angeles, CA, USA.
  • Heikkinen T; CHDI Management/CHDI Foundation, Los Angeles, CA, USA.
  • Lehtimäki KK; CHDI Management/CHDI Foundation, Los Angeles, CA, USA.
  • Svedberg MM; Charles River Discovery Services, Kuopio, Finland.
  • Häggkvist J; Charles River Discovery Services, Kuopio, Finland.
  • Tari L; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden.
  • Tóth M; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden.
  • Varrone A; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden.
  • Halldin C; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden.
  • Kudwa AE; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden.
  • Ramboz S; Department of Clinical Neuroscience, Center for Psychiatry Research, Karolinska Institutet and Stockholm County Council, Stockholm, Sweden.
  • Day M; Psychogenics, Inc., Paramus, NJ, USA.
  • Kondapalli J; Psychogenics, Inc., Paramus, NJ, USA.
  • Surmeier DJ; Department of Physiology, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.
  • Urnov FD; Department of Physiology, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.
  • Gregory PD; Department of Physiology, Feinberg School of Medicine, Northwestern University, Chicago, IL, USA.
  • Rebar EJ; Sangamo Therapeutics, Inc., Richmond, CA, USA.
  • Muñoz-Sanjuán I; Innovative Genomics Institute, Berkeley, CA, USA.
Nat Med ; 25(7): 1131-1142, 2019 07.
Article em En | MEDLINE | ID: mdl-31263285
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transcrição Gênica / Dedos de Zinco / Doença de Huntington / Alelos / Proteína Huntingtina / Mutação Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Revista: Nat Med Assunto da revista: BIOLOGIA MOLECULAR / MEDICINA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transcrição Gênica / Dedos de Zinco / Doença de Huntington / Alelos / Proteína Huntingtina / Mutação Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Revista: Nat Med Assunto da revista: BIOLOGIA MOLECULAR / MEDICINA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Estados Unidos