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Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) ß-thalassemia mutation.
Hardouin, Giulia; Antoniou, Panagiotis; Martinucci, Pierre; Felix, Tristan; Manceau, Sandra; Joseph, Laure; Masson, Cécile; Scaramuzza, Samantha; Ferrari, Giuliana; Cavazzana, Marina; Miccio, Annarita.
Afiliación
  • Hardouin G; Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, INSERM UMR1163, Paris Cité University, Paris, France.
  • Antoniou P; Biotherapy Clinical Investigation Center, Necker Children's Hospital, Assistance Publique Hopitaux de Paris, Paris, France.
  • Martinucci P; Human Lymphohematopoiesis Laboratory, Imagine Institute, INSERM UMR1163, Paris Cité University, Paris, France.
  • Felix T; Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, INSERM UMR1163, Paris Cité University, Paris, France.
  • Manceau S; Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, INSERM UMR1163, Paris Cité University, Paris, France.
  • Joseph L; Laboratory of Chromatin and Gene Regulation during Development, Imagine Institute, INSERM UMR1163, Paris Cité University, Paris, France.
  • Masson C; Biotherapy Clinical Investigation Center, Necker Children's Hospital, Assistance Publique Hopitaux de Paris, Paris, France.
  • Scaramuzza S; Biotherapy Clinical Investigation Center, Necker Children's Hospital, Assistance Publique Hopitaux de Paris, Paris, France.
  • Ferrari G; Biotherapy Department, Necker Children's Hospital, Assistance Publique Hopitaux de Paris, Paris, France.
  • Cavazzana M; Bioinformatics Platform, Imagine Institute, INSERM UMR1163, Paris Cité University, Paris, France.
  • Miccio A; San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific Institute, Milan, Italy.
Blood ; 141(10): 1169-1179, 2023 03 09.
Article en En | MEDLINE | ID: mdl-36508706

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Talasemia beta Límite: Humans Idioma: En Revista: Blood Año: 2023 Tipo del documento: Article País de afiliación: Francia

Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Talasemia beta Límite: Humans Idioma: En Revista: Blood Año: 2023 Tipo del documento: Article País de afiliación: Francia