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DNA Base Editing Could Reverse Most Disease-Causing Point Mutations

DNA Base Editing Could Reverse Most Disease-Causing Point Mutations Most of the more than 50 000 genetic changes currently associated with human diseases are point mutations, the swap of one DNA base pair for another. Now, researchers at Harvard University and the Broad Institute have developed a genome editing technology called base editing that changes one DNA base pair to another, potentially allowing for curative point mutation corrections. Writing in Nature in October, the researchers described adenine base editors that efficiently converted target A · T base pairs to G · C pairs in bacteria and human cells with almost no undesired or unintended modifications. The same team first introduced base editing last year with a technique that switches C · G base pairs into T · A pairs. Together, the methods could 1 day enable the reversal or suppression of most disease-causing point mutations, including those associated with certain types of sickle cell anemia, hemophilia, cystic fibrosis, phenylketonuria, and amyotrophic lateral sclerosis, among many other conditions. Base editing uses a modified form of the CRISPR-Cas9 gene editing system that does not make double-stranded cuts in DNA, explained David R. Liu, PhD, a professor of chemistry and chemical biology at Harvard University, vice-chair of the faculty at the Broad, and a Howard Hughes Medical Institute investigator. “Standard genome editing methods make double-stranded breaks in DNA, which is especially useful when the goal is to insert or delete DNA bases,” he said. “But when the goal is simply to fix a point mutation, base editing offers a more efficient and substantially cleaner solution.” Much additional work is needed to translate base editors into human therapeutics, Liu said, including pairing them with effective delivery solutions and testing their safety and efficacy in animal models. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA American Medical Association

DNA Base Editing Could Reverse Most Disease-Causing Point Mutations

Abbasi, Jennifer
JAMA , Volume 318 (22) – Dec 12, 2017
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DNA Base Editing Could Reverse Most Disease-Causing Point Mutations

Abstract

Most of the more than 50 000 genetic changes currently associated with human diseases are point mutations, the swap of one DNA base pair for another. Now, researchers at Harvard University and the Broad Institute have developed a genome editing technology called base editing that changes one DNA base pair to another, potentially allowing for curative point mutation corrections. Writing in Nature in October, the researchers described adenine base editors that efficiently converted target...
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Publisher
American Medical Association
Copyright
Copyright © 2017 American Medical Association. All Rights Reserved.
ISSN
0098-7484
eISSN
1538-3598
DOI
10.1001/jama.2017.19063
Publisher site
See Article on Publisher Site

Abstract

Most of the more than 50 000 genetic changes currently associated with human diseases are point mutations, the swap of one DNA base pair for another. Now, researchers at Harvard University and the Broad Institute have developed a genome editing technology called base editing that changes one DNA base pair to another, potentially allowing for curative point mutation corrections. Writing in Nature in October, the researchers described adenine base editors that efficiently converted target A · T base pairs to G · C pairs in bacteria and human cells with almost no undesired or unintended modifications. The same team first introduced base editing last year with a technique that switches C · G base pairs into T · A pairs. Together, the methods could 1 day enable the reversal or suppression of most disease-causing point mutations, including those associated with certain types of sickle cell anemia, hemophilia, cystic fibrosis, phenylketonuria, and amyotrophic lateral sclerosis, among many other conditions. Base editing uses a modified form of the CRISPR-Cas9 gene editing system that does not make double-stranded cuts in DNA, explained David R. Liu, PhD, a professor of chemistry and chemical biology at Harvard University, vice-chair of the faculty at the Broad, and a Howard Hughes Medical Institute investigator. “Standard genome editing methods make double-stranded breaks in DNA, which is especially useful when the goal is to insert or delete DNA bases,” he said. “But when the goal is simply to fix a point mutation, base editing offers a more efficient and substantially cleaner solution.” Much additional work is needed to translate base editors into human therapeutics, Liu said, including pairing them with effective delivery solutions and testing their safety and efficacy in animal models.

Journal

JAMA – American Medical Association

Published: Dec 12, 2017

Keywords: biotechnology,dna,point mutation

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