Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells.

Omer-Javed, Attya; Pedrazzani, Gabriele; Albano, Luisa; Ghaus, Sherash; Latroche, Claire; Manzi, Maura; Ferrari, Samuele; Fiumara, Martina; Jacob, Aurelien; Vavassori, Valentina; Nonis, Alessandro; Canarutto, Daniele; Naldini, Luigi

Omer-Javed, Attya; Pedrazzani, Gabriele; Albano, Luisa; Ghaus, Sherash; Latroche, Claire; Manzi, Maura; Ferrari, Samuele; Fiumara, Martina; Jacob, Aurelien; Vavassori, Valentina; Nonis, Alessandro; Canarutto, Daniele; Naldini, Luigi.

Afiliação

Omer-Javed A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Pedrazzani G; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy; Vita-Salute San Raffaele University, Milan 20132, Italy.
Albano L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Ghaus S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Latroche C; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Manzi M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Ferrari S; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Fiumara M; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy; Vita-Salute San Raffaele University, Milan 20132, Italy.
Jacob A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Vavassori V; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.
Nonis A; CUSSB-University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan, Italy.
Canarutto D; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy; Vita-Salute San Raffaele University, Milan 20132, Italy; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Naldini L; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy; Vita-Salute San Raffaele University, Milan 20132, Italy. Electronic address: naldini.luigi@hsr.it.

Cell ; 185(13): 2248-2264.e21, 2022 06 23.

Article em En | MEDLINE | ID: mdl-35617958

Assuntos

Edição de Genes; Transplante de Células-Tronco Hematopoéticas; Animais; Terapia Genética/métodos; Transplante de Células-Tronco Hematopoéticas/métodos; Células-Tronco Hematopoéticas; Humanos; Camundongos

Palavras-chave

CRISPR-Cas gene editing; RNA-based delivery; X-linked hyper-IgM syndrome; autologous stem cell transplantation; conditioning-free; gene transfer; hematopoietic stem cells; human hematochimeric mouse model; mobilization

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Edição de Genes Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

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