Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34<sup>+</sup> cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.

Liu, Yang; Dahl, Maria; Debnath, Shubhranshu; Rothe, Michael; Smith, Emma M; Grahn, Tan Hooi Min; Warsi, Sarah; Chen, Jun; Flygare, Johan; Schambach, Axel; Karlsson, Stefan

Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34⁺ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.

Liu, Yang; Dahl, Maria; Debnath, Shubhranshu; Rothe, Michael; Smith, Emma M; Grahn, Tan Hooi Min; Warsi, Sarah; Chen, Jun; Flygare, Johan; Schambach, Axel; Karlsson, Stefan.

Afiliación

Liu Y; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184. yang.liu@med.lu.se.
Dahl M; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184.
Debnath S; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184.
Rothe M; Institute of Experimental Hematology, Hannover Medical School, Hannover 30625.
Smith EM; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184.
Grahn THM; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184.
Warsi S; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184.
Chen J; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184.
Flygare J; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184.
Schambach A; Institute of Experimental Hematology, Hannover Medical School, Hannover 30625, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston.
Karlsson S; Molecular Medicine and Gene Therapy, Lund Stem Cell Center, Lund University, Lund 22184. stefan.karlsson@med.lu.se.

Haematologica ; 107(2): 446-456, 2022 02 01.

Article en En | MEDLINE | ID: mdl-33440921

Asunto(s)

Anemia de Diamond-Blackfan; Anemia de Diamond-Blackfan/genética; Anemia de Diamond-Blackfan/metabolismo; Anemia de Diamond-Blackfan/terapia; Animales; Sangre Fetal/metabolismo; Terapia Genética; Células Madre Hematopoyéticas/metabolismo; Humanos; Ratones; Mutación; ARN Interferente Pequeño/genética; Proteínas Ribosómicas/genética

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Anemia de Diamond-Blackfan Tipo de estudio: Prognostic_studies Límite: Animals / Humans Idioma: En Revista: Haematologica Año: 2022 Tipo del documento: Article

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