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Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities.
Khoja, Suhail; Lambert, Jenna; Nitzahn, Matthew; Eliav, Adam; Zhang, YuChen; Tamboline, Mikayla; Le, Colleen T; Nasser, Eram; Li, Yunfeng; Patel, Puja; Zhuravka, Irina; Lueptow, Lindsay M; Tkachyova, Ilona; Xu, Shili; Nissim, Itzhak; Schulze, Andreas; Lipshutz, Gerald S.
Afiliação
  • Khoja S; Department of Surgery, UCLA, Los Angeles, CA 90025, USA.
  • Lambert J; Department of Surgery, UCLA, Los Angeles, CA 90025, USA.
  • Nitzahn M; Molecular Biology Institute, UCLA, Los Angeles, CA 90025, USA.
  • Eliav A; Department of Surgery, UCLA, Los Angeles, CA 90025, USA.
  • Zhang Y; Semel Institute for Neuroscience, UCLA, Los Angeles, CA 90025, USA.
  • Tamboline M; Crump Institute for Molecular Imaging, UCLA, Los Angeles, CA 90025, USA.
  • Le CT; Departments of Molecular and Medical Pharmacology, Universtiy of California, Los Angeles, CA 90025, USA.
  • Nasser E; Department of Surgery, UCLA, Los Angeles, CA 90025, USA.
  • Li Y; Department of Surgery, UCLA, Los Angeles, CA 90025, USA.
  • Patel P; Departments of Pathology and Laboratory Medicine, UCLA, Los Angeles, CA 90025, USA.
  • Zhuravka I; Department of Surgery, UCLA, Los Angeles, CA 90025, USA.
  • Lueptow LM; Behavioral Testing Core, Department of Psychology, UCLA, Los Angeles, CA 90025, USA.
  • Tkachyova I; Behavioral Testing Core, Department of Psychology, UCLA, Los Angeles, CA 90025, USA.
  • Xu S; Research Institute, The Hospital for Sick Children, Toronto, ON M5G 1X8, Canada.
  • Nissim I; Crump Institute for Molecular Imaging, UCLA, Los Angeles, CA 90025, USA.
  • Schulze A; Departments of Molecular and Medical Pharmacology, Universtiy of California, Los Angeles, CA 90025, USA.
  • Lipshutz GS; Jonsson Comprehensive Cancer Center at UCLA, David Geffen School of Medicine at UCLA, Los Angeles, CA 90025, USA.
Mol Ther Methods Clin Dev ; 25: 278-296, 2022 Jun 09.
Article em En | MEDLINE | ID: mdl-35505663
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Prognostic_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2022 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 Tipo de estudo: Prognostic_studies Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos