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The New England Journal of Medicine, ISSN 0028-4793, 03/2019, Volume 380, Issue 10, pp. 947 - 959
Journal Article
2016, 1st ed. 2016, Advances in experimental medicine and biology, ISBN 9781493935079, Volume 895, xvi, 263 pages
This comprehensive volume explores human genetic engineering its pre-clinical and clinical applications, current developments, and as treatment for hereditary... 
Gene therapy | Genetic engineering | Human genetics | Human Genetics | Biomedicine
Book
Advances in Experimental Medicine and Biology, ISSN 0065-2598, 2017, Volume 1013, pp. 203 - 217
The β-hemoglobinopathies are diverse set of disorders caused by mutations in the β-globin (HBB) gene. Because HBB protein is a critical component (along with... 
Nucleases | Genome editing | Sickle cell disease | β-hemoglobinopathies | HBB gene | β-thalassemia
Journal Article
Molecular Therapy, ISSN 1525-0016, 2013, Volume 21, Issue 4, pp. 786 - 795
Journal Article
Molecular Therapy, ISSN 1525-0016, 05/2016, Volume 24, pp. S225 - S225
Journal Article
Journal Article
Blood, ISSN 0006-4971, 12/2014, Volume 124, Issue 21, pp. 883 - 883
Abstract Chromosomal rearrangements involving the MLL gene occur in both primary and treatment-related leukemias, and are associated with a poor prognosis.... 
Journal Article
Science, ISSN 0036-8075, 10/2013, Volume 342, Issue 6155, pp. 253 - 257
Journal Article
Blood, ISSN 0006-4971, 05/2016, Volume 127, Issue 21, pp. 2507 - 2509
In this issue of Blood , Hubbard et al provide important proof-of-concept data on the utility of using genome editing to knock-in a wild-type (WT)... 
HEMATOLOGY | CELLS | GENOME | RNA Editing | Humans | Gene Editing | CRISPR-Cas Systems | Inside
Journal Article