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Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome.
Shao, Yingyao; Sztainberg, Yehezkel; Wang, Qi; Bajikar, Sameer S; Trostle, Alexander J; Wan, Ying-Wooi; Jafar-Nejad, Paymaan; Rigo, Frank; Liu, Zhandong; Tang, Jianrong; Zoghbi, Huda Y.
Afiliação
  • Shao Y; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Sztainberg Y; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
  • Wang Q; Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030, USA.
  • Bajikar SS; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Trostle AJ; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
  • Wan YW; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
  • Jafar-Nejad P; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Rigo F; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Liu Z; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
  • Tang J; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, Houston, TX 77030, USA.
  • Zoghbi HY; Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
Sci Transl Med ; 13(583)2021 03 03.
Article em En | MEDLINE | ID: mdl-33658357

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Deficiência Intelectual Ligada ao Cromossomo X / Proteína 2 de Ligação a Metil-CpG Limite: Animals Idioma: En Revista: Sci Transl Med Assunto da revista: CIENCIA / MEDICINA 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: Oligonucleotídeos Antissenso / Deficiência Intelectual Ligada ao Cromossomo X / Proteína 2 de Ligação a Metil-CpG Limite: Animals Idioma: En Revista: Sci Transl Med Assunto da revista: CIENCIA / MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos