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A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy.
Antony, Justin S; Daniel-Moreno, Alberto; Lamsfus-Calle, Andrés; Raju, Janani; Kaftancioglu, Merve; Ureña-Bailén, Guillermo; Rottenberger, Jennifer; Hou, Yujuan; Santhanakumaran, Vidiyaah; Lee, Jun-Hoe; Heumos, Lukas; Böhringer, Judith; Krägeloh-Mann, Ingeborg; Handgretinger, Rupert; Mezger, Markus.
Afiliação
  • Antony JS; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Daniel-Moreno A; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Lamsfus-Calle A; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Raju J; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Kaftancioglu M; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Ureña-Bailén G; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Rottenberger J; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Hou Y; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Santhanakumaran V; Department of Pediatric Neurology, University Children's Hospital Tübingen, Germany.
  • Lee JH; Quantitative Biology Center (QBiC), University of Tübingen, Tübingen, Germany.
  • Heumos L; Quantitative Biology Center (QBiC), University of Tübingen, Tübingen, Germany.
  • Böhringer J; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
  • Krägeloh-Mann I; Department of Pediatric Neurology, University Children's Hospital Tübingen, Germany.
  • Handgretinger R; Department of Pediatric Neurology, University Children's Hospital Tübingen, Germany.
  • Mezger M; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.
CRISPR J ; 5(1): 66-79, 2022 02.
Article em En | MEDLINE | ID: mdl-34882002
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Texto completo: 1 Coleções: 01-internacional Contexto em Saúde: 6_ODS3_enfermedades_notrasmisibles Base de dados: MEDLINE Assunto principal: Leucodistrofia Metacromática Tipo de estudo: Observational_studies Limite: Humans Idioma: En Revista: CRISPR J Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Contexto em Saúde: 6_ODS3_enfermedades_notrasmisibles Base de dados: MEDLINE Assunto principal: Leucodistrofia Metacromática Tipo de estudo: Observational_studies Limite: Humans Idioma: En Revista: CRISPR J Ano de publicação: 2022 Tipo de documento: Article