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A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis.
Kell, Pamela; Sidhu, Rohini; Qian, Mingxing; Mishra, Sonali; Nicoli, Elena-Raluca; D'Souza, Precilla; Tifft, Cynthia J; Gross, Amanda L; Gray-Edwards, Heather L; Martin, Douglas R; Sena-Esteves, Miguel; Dietzen, Dennis J; Singh, Manmilan; Luo, Jingqin; Schaffer, Jean E; Ory, Daniel S; Jiang, Xuntian.
Affiliation
  • Kell P; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Sidhu R; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Qian M; Department of Developmental Biology, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Mishra S; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Nicoli ER; Medical Genetics Branch and Office of the Clinical Director, NHGRI, NIH, Bethesda, MD 20892, USA.
  • D'Souza P; Medical Genetics Branch and Office of the Clinical Director, NHGRI, NIH, Bethesda, MD 20892, USA; Office of the Clinical Director, NHGRI, NIH, Bethesda, MD, 20892, USA.
  • Tifft CJ; Medical Genetics Branch and Office of the Clinical Director, NHGRI, NIH, Bethesda, MD 20892, USA; Office of the Clinical Director, NHGRI, NIH, Bethesda, MD, 20892, USA.
  • Gross AL; Scott-Ritchey Research Center, Auburn University College of Veterinary Medicine, Auburn, AL, 36849, USA.
  • Gray-Edwards HL; Scott-Ritchey Research Center, Auburn University College of Veterinary Medicine, Auburn, AL, 36849, USA.
  • Martin DR; Scott-Ritchey Research Center, Auburn University College of Veterinary Medicine, Auburn, AL, 36849, USA.
  • Sena-Esteves M; Department of Neurology, Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
  • Dietzen DJ; Department of Pathology & Immunology, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Singh M; Department of Chemistry, Washington University, St. Louis, MO, 63130, USA.
  • Luo J; Department of Surgery, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Schaffer JE; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Ory DS; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Jiang X; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA. Electronic address: jiangxuntian@wustl.edu.
EBioMedicine ; 92: 104627, 2023 Jun.
Article de En | MEDLINE | ID: mdl-37267847
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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Gangliosidose à GM1 / Maladies neurodégénératives Type d'étude: Prognostic_studies Limites: Animals Langue: En Journal: EBioMedicine Année: 2023 Type de document: Article Pays d'affiliation: États-Unis d'Amérique
Texte intégral
Ajouter à My VHL
Imprimer
XML
PubMed Links
Recherche sur Google

Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Gangliosidose à GM1 / Maladies neurodégénératives Type d'étude: Prognostic_studies Limites: Animals Langue: En Journal: EBioMedicine Année: 2023 Type de document: Article Pays d'affiliation: États-Unis d'Amérique