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Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches

Autor: Lamsfus-Calle, Andrés, Daniel-Moreno, Alberto, Ureña-Bailén, Guillermo, Raju, Janani, Antony, Justin S., Handgretinger, Rupert, Mezger, Markus

Publikováno v: In Blood Reviews March 2020 40

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A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy.

Autor: 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.; Department of Pediatric Neurology, University Children's Hospital Tübingen, Germany., Krägeloh-Mann I; Department of Pediatric Neurology, University Children's Hospital Tübingen, Germany., Handgretinger R; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany., Mezger M; Department of Hematology and Oncology, University Children's Hospital, University of Tübingen, Germany.

Publikováno v: The CRISPR journal [CRISPR J] 2022 Feb; Vol. 5 (1), pp. 66-79. Date of Electronic Publication: 2021 Dec 08.