Abstract:
:Despite the recent discover of genome-editing methods, today we can say these approaches have firmly entered our life. Two approaches-knocking out malfunctioning gene allele or correcting the mutation with precise knock-in-can be used in hereditary monogenic diseases treatment. The latter approach is relatively ineffective. Modern data about the ways of repair of double-strand DNA breaks formed by nucleases are presented in this review. The main part of the review is devoted to the ways of increasing precise and effective knock-in: inhibition of non-homologous end joining and stimulation of homology-directed repair key factors, use of small molecules with unknown mechanism of action, cell-cycle synchronization and cell-cycle-dependent activity of Cas9, donor molecule design, selection, alternative methods for insertion and other approaches.
journal_name
Hum Genetjournal_title
Human geneticsauthors
Smirnikhina SA,Anuchina AA,Lavrov AVdoi
10.1007/s00439-018-1953-5subject
Has Abstractpub_date
2019-01-01 00:00:00pages
1-19issue
1eissn
0340-6717issn
1432-1203pii
10.1007/s00439-018-1953-5journal_volume
138pub_type
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