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Gene activation of SMN by selective disruption of lncRNA-mediated recruitment of PRC2 for the treatment of spinal muscular atrophy.
Woo, Caroline J; Maier, Verena K; Davey, Roshni; Brennan, James; Li, Guangde; Brothers, John; Schwartz, Brian; Gordo, Susana; Kasper, Anne; Okamoto, Trevor R; Johansson, Hans E; Mandefro, Berhan; Sareen, Dhruv; Bialek, Peter; Chau, B Nelson; Bhat, Balkrishen; Bullough, David; Barsoum, James.
Afiliação
  • Woo CJ; RaNA Therapeutics, Cambridge, MA 02139; cwoo@ranarx.com.
  • Maier VK; RaNA Therapeutics, Cambridge, MA 02139.
  • Davey R; RaNA Therapeutics, Cambridge, MA 02139.
  • Brennan J; RaNA Therapeutics, Cambridge, MA 02139.
  • Li G; RaNA Therapeutics, Cambridge, MA 02139.
  • Brothers J; RaNA Therapeutics, Cambridge, MA 02139.
  • Schwartz B; RaNA Therapeutics, Cambridge, MA 02139.
  • Gordo S; RaNA Therapeutics, Cambridge, MA 02139.
  • Kasper A; RaNA Therapeutics, Cambridge, MA 02139.
  • Okamoto TR; LGC Biosearch Technologies, Petaluma, CA 94954.
  • Johansson HE; LGC Biosearch Technologies, Petaluma, CA 94954.
  • Mandefro B; Board of Governors-Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA 90048.
  • Sareen D; Induced Pluripotent Stem Cell Core, The David and Janet Polak Stem Cell Laboratory, Cedars-Sinai Medical Center, Los Angeles, CA 90048.
  • Bialek P; Board of Governors-Regenerative Medicine Institute, Cedars-Sinai Medical Center, Los Angeles, CA 90048.
  • Chau BN; Induced Pluripotent Stem Cell Core, The David and Janet Polak Stem Cell Laboratory, Cedars-Sinai Medical Center, Los Angeles, CA 90048.
  • Bhat B; Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, CA 90048.
  • Bullough D; RaNA Therapeutics, Cambridge, MA 02139.
  • Barsoum J; RaNA Therapeutics, Cambridge, MA 02139.
Proc Natl Acad Sci U S A ; 114(8): E1509-E1518, 2017 02 21.
Article em En | MEDLINE | ID: mdl-28193854
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Atrofia Muscular Espinal / Proteína 2 de Sobrevivência do Neurônio Motor / Complexo Repressor Polycomb 2 / RNA Longo não Codificante Limite: Animals / Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Atrofia Muscular Espinal / Proteína 2 de Sobrevivência do Neurônio Motor / Complexo Repressor Polycomb 2 / RNA Longo não Codificante Limite: Animals / Humans Idioma: En Ano de publicação: 2017 Tipo de documento: Article