Preclinical evaluation for engraftment of CD34<sup>+</sup> cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models.

Uchida, Naoya; Li, Linhong; Nassehi, Tina; Drysdale, Claire M; Yapundich, Morgan; Gamer, Jackson; Haro-Mora, Juan J; Demirci, Selami; Leonard, Alexis; Bonifacino, Aylin C; Krouse, Allen E; Linde, N Seth; Allen, Cornell; Peshwa, Madhusudan V; De Ravin, Suk See; Donahue, Robert E; Malech, Harry L; Tisdale, John F

Preclinical evaluation for engraftment of CD34⁺ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models.

Uchida, Naoya; Li, Linhong; Nassehi, Tina; Drysdale, Claire M; Yapundich, Morgan; Gamer, Jackson; Haro-Mora, Juan J; Demirci, Selami; Leonard, Alexis; Bonifacino, Aylin C; Krouse, Allen E; Linde, N Seth; Allen, Cornell; Peshwa, Madhusudan V; De Ravin, Suk See; Donahue, Robert E; Malech, Harry L; Tisdale, John F.

Afiliação

Uchida N; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Li L; Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo, Japan.
Nassehi T; MaxCyte, Gaithersburg, MD, USA.
Drysdale CM; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Yapundich M; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Gamer J; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Haro-Mora JJ; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Demirci S; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Leonard A; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Bonifacino AC; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Krouse AE; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA.
Linde NS; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA.
Allen C; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA.
Peshwa MV; MaxCyte, Gaithersburg, MD, USA.
De Ravin SS; MaxCyte, Gaithersburg, MD, USA.
Donahue RE; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), NIH, Bethesda, MD, USA.
Malech HL; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Tisdale JF; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), NIH, Bethesda, MD, USA.

Cell Rep Med ; 2(4): 100247, 2021 04 20.

Article em En | MEDLINE | ID: mdl-33948577

Assuntos

Anemia Falciforme/genética; Antígenos CD34/metabolismo; Células-Tronco Hematopoéticas/metabolismo; Xenoenxertos/imunologia; Macaca mulatta/genética; Animais; Edição de Genes/métodos; Marcação de Genes/métodos; Transplante de Células-Tronco Hematopoéticas/métodos; Hemoglobina Falciforme/genética; Humanos; Camundongos; RNA Guia de Cinetoplastídeos/metabolismo; Globinas beta/genética

Palavras-chave

CRISPR/Cas9; electroporation; gene correction; genome editing; hematopoietic stem cell; large animal model; non-human primate; sickle cell disease; transplantation; ß-globin gene

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Células-Tronco Hematopoéticas / Antígenos CD34 / Xenoenxertos / Anemia Falciforme / Macaca mulatta Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Cell Rep Med Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo

Imprimir

XML

PubMed Links

Buscar no Google