Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements.

Wang, Hongjie; Georgakopoulou, Aphrodite; Li, Chang; Liu, Zhinan; Gil, Sucheol; Bashyam, Ashvin; Yannaki, Evangelia; Anagnostopoulos, Achilles; Pande, Amit; Izsvák, Zsuzsanna; Papayannopoulou, Thalia; Lieber, André

Wang, Hongjie; Georgakopoulou, Aphrodite; Li, Chang; Liu, Zhinan; Gil, Sucheol; Bashyam, Ashvin; Yannaki, Evangelia; Anagnostopoulos, Achilles; Pande, Amit; Izsvák, Zsuzsanna; Papayannopoulou, Thalia; Lieber, André.

Afiliación

Wang H; Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA.
Georgakopoulou A; Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA.
Li C; Hematology Department, Hematopoietic Stem Cell Transplantation Unit, George Papanicolaou Hospital, Thessaloniki, Greece.
Liu Z; Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA.
Gil S; Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA.
Bashyam A; Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA.
Yannaki E; 5AM Ventures, Boston, Massachusetts, USA.
Anagnostopoulos A; Hematology Department, Hematopoietic Stem Cell Transplantation Unit, George Papanicolaou Hospital, Thessaloniki, Greece.
Pande A; Hematology Department, Hematopoietic Stem Cell Transplantation Unit, George Papanicolaou Hospital, Thessaloniki, Greece.
Izsvák Z; Max Delbrück Center for Molecular Medicine in the Helmholtz Association, Berlin, Germany.
Papayannopoulou T; Max Delbrück Center for Molecular Medicine in the Helmholtz Association, Berlin, Germany.
Lieber A; Division of Hematology, Department of Medicine, and.

JCI Insight ; 5(16)2020 08 20.

Article en En | MEDLINE | ID: mdl-32814708

Asunto(s)

Terapia Genética/métodos; Vectores Genéticos/genética; Células Madre Hematopoyéticas/fisiología; Secuencias Reguladoras de Ácidos Nucleicos/genética; Talasemia/genética; Adenoviridae/genética; Animales; Antígenos CD34/metabolismo; Elementos Transponibles de ADN; Modelos Animales de Enfermedad; Femenino; Trasplante de Células Madre Hematopoyéticas/métodos; Humanos; Proteína Cofactora de Membrana/genética; Ratones Endogámicos C57BL; Ratones Transgénicos; Talasemia/terapia; Transducción Genética; Globinas beta/genética; gamma-Globinas/genética

Palabras clave

Gene therapy; Hematology; Hematopoietic stem cells; Mouse models; Therapeutics

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Talasemia / Células Madre Hematopoyéticas / Terapia Genética / Secuencias Reguladoras de Ácidos Nucleicos / Vectores Genéticos Límite: Animals / Female / Humans Idioma: En Revista: JCI Insight Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos

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