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Science, ISSN 0036-8075, 11/2014, Volume 346, Issue 6212, pp. 987 - 991
Journal Article
Blood, ISSN 0006-4971, 11/2019, Volume 134, Issue Supplement_1, pp. 2246 - 2246
Despite the significant advances in developing novel therapies for hemoglobinopathies, developing a universal and robust therapeutic approach, able to achieve... 
Journal Article
Journal Article
Nucleic Acids Research, ISSN 0305-1048, 01/2006, Volume 34, Issue 1, pp. e2 - e2
Journal Article
BLOOD, ISSN 0006-4971, 06/2018, Volume 131, Issue 26, pp. 2915 - 2928
Disorders involving beta-globin gene mutations, primarily beta-thalassemia and sickle cell disease, represent a major target for hematopoietic stem/progenitor... 
CD34(+) CELLS | INTRAVENOUS-INJECTION | BCL11A | DISEASE | FETAL-HEMOGLOBIN EXPRESSION | GENE-EXPRESSION | ADENOVIRAL VECTORS | HEREDITARY PERSISTENCE | HEMATOLOGY | EFFICIENT | BLOOD-CELLS | Gene Therapy
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Molecular Therapy, ISSN 1525-0016, 04/2004, Volume 9, Issue 4, pp. 496 - 509
Journal Article