CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

Two patients, one with transfusion-dependent β-thalassemia and the other with sickle cell disease, received autologous CD34+ cells edited with CRISPR-Cas9 targeting of BCL11A . Their clinical course over the following 16 to 18 months supports further experimental testing of CRISPR-Cas9 gene editing...

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Veröffentlicht in:	The New England journal of medicine 2021-01, Vol.384 (3), p.252-260
Hauptverfasser:	Frangoul, Haydar, Altshuler, David, Cappellini, M. Domenica, Chen, Yi-Shan, Domm, Jennifer, Eustace, Brenda K, Foell, Juergen, de la Fuente, Josu, Grupp, Stephan, Handgretinger, Rupert, Ho, Tony W, Kattamis, Antonis, Kernytsky, Andrew, Lekstrom-Himes, Julie, Li, Amanda M, Locatelli, Franco, Mapara, Markus Y, de Montalembert, Mariane, Rondelli, Damiano, Sharma, Akshay, Sheth, Sujit, Soni, Sandeep, Steinberg, Martin H, Wall, Donna, Yen, Angela, Corbacioglu, Selim
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Anemia, Sickle Cell - genetics Anemia, Sickle Cell - therapy Autografts beta-Thalassemia - genetics beta-Thalassemia - therapy Bone marrow CD34 antigen CRISPR CRISPR-Cas Systems Deoxyribonucleic acid DNA DNA nucleotidylexotransferase Electroporation Erythroid cells Female Fetal Hemoglobin - biosynthesis Fetal Hemoglobin - genetics Fetuses Gene Editing - methods General & Internal Medicine Genetic Therapy Genome editing Genotype & phenotype Hematopoietic stem cells Hemoglobin Humans Iron Laboratories Life Sciences & Biomedicine Medicine, General & Internal Mutation Patients Progenitor cells Repressor Proteins - genetics Repressor Proteins - metabolism Science & Technology Sickle cell disease Thalassemia Young Adult
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Beschreibung
Zusammenfassung:	Two patients, one with transfusion-dependent β-thalassemia and the other with sickle cell disease, received autologous CD34+ cells edited with CRISPR-Cas9 targeting of BCL11A . Their clinical course over the following 16 to 18 months supports further experimental testing of CRISPR-Cas9 gene editing to treat these diseases.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2031054