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The promise and challenge of therapeutic genome editing

Nature (London), 2020-02, Vol.578 (7794), p.229-236 [Peer Reviewed Journal]

COPYRIGHT 2020 Nature Publishing Group ;COPYRIGHT 2020 Nature Publishing Group ;Copyright Nature Publishing Group Feb 13, 2020 ;ISSN: 0028-0836 ;EISSN: 1476-4687 ;DOI: 10.1038/s41586-020-1978-5 ;PMID: 32051598

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Title:
The promise and challenge of therapeutic genome editing
Author: Doudna, Jennifer A
Subjects: Anemia, Sickle Cell - genetics ; Anemia, Sickle Cell - therapy ; beta-Globins - genetics ; Blood ; CRISPR ; CRISPR-Cas Systems - genetics ; Deoxyribonucleic acid ; DNA ; DNA sequencing ; Editing ; Enzymes ; Forecasts and trends ; Gene Editing - ethics ; Gene Editing - methods ; Gene Editing - standards ; Gene Editing - trends ; Gene sequencing ; Gene therapy ; Genetic disorders ; Genetic engineering ; Genetics ; Genome, Human - genetics ; Genomes ; Genomics ; Germ-Line Mutation - genetics ; Health aspects ; Hemoglobin ; Humans ; Methods ; Muscular dystrophy ; Muscular Dystrophy, Duchenne - genetics ; Muscular Dystrophy, Duchenne - therapy ; Mutation ; Nucleotide sequence ; Nucleotide sequencing ; Organ Specificity - genetics ; Patient Safety ; Patients ; Proteins ; Sickle cell disease ; Stem cells ; Technology ; Technology application ; Therapeutic applications
Is Part Of: Nature (London), 2020-02, Vol.578 (7794), p.229-236
Description: Genome editing, which involves the precise manipulation of cellular DNA sequences to alter cell fates and organism traits, has the potential to both improve our understanding of human genetics and cure genetic disease. Here I discuss the scientific, technical and ethical aspects of using CRISPR (clustered regularly interspaced short palindromic repeats) technology for therapeutic applications in humans, focusing on specific examples that highlight both opportunities and challenges. Genome editing is-or will soon be-in the clinic for several diseases, with more applications under development. The rapid pace of the field demands active efforts to ensure that this breakthrough technology is used responsibly to treat, cure and prevent genetic disease.
Publisher: England: Nature Publishing Group
Language: English
Identifier: ISSN: 0028-0836
EISSN: 1476-4687
DOI: 10.1038/s41586-020-1978-5
PMID: 32051598
Source: ProQuest One Psychology
AUTh Library subscriptions: ProQuest Central
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The promise and challenge of therapeutic genome editing

COPYRIGHT 2020 Nature Publishing Group ;COPYRIGHT 2020 Nature Publishing Group ;Copyright Nature Publishing Group Feb 13, 2020 ;ISSN: 0028-0836 ;EISSN: 1476-4687 ;DOI: 10.1038/s41586-020-1978-5 ;PMID: 32051598

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