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Genome-editing technologies for gene correction of hemophilia

Authors
 Chul?Yong Park  ;  Dongjin R. Lee  ;  Jin Jea Sung  ;  Dong?Wook Kim 
Citation
 HUMAN GENETICS, Vol.135(9) : 977-981, 2016 
Journal Title
HUMAN GENETICS
ISSN
 0340-6717 
Issue Date
2016
MeSH
Gene Editing* ; Genetic Therapy* ; Hemophilia A/therapy* ; Humans
Abstract
Hemophilia is caused by various mutations in blood coagulation factor genes, including factor VIII (FVIII) and factor IX (FIX), that encode key proteins in the blood clotting pathway. Although the addition of therapeutic genes or infusion of clotting factors may be used to remedy hemophilia's symptoms, no permanent cure for the disease exists. Moreover, patients often develop neutralizing antibodies or experience adverse effects that limit the therapy's benefits. However, targeted gene therapy involving the precise correction of these mutated genes at the genome level using programmable nucleases is a promising strategy. These nucleases can induce double-strand breaks (DSBs) on genomes, and repairs of such induced DSBs by the two cellular repair systems enable a targeted gene correction. Going beyond cultured cell systems, we are now entering the age of direct gene correction in vivo using various delivery tools. Here, we describe the current status of in vivo and ex vivo genome-editing technology related to potential hemophilia gene correction and the prominent issues surrounding its application in patients with monogenic diseases.
Files in This Item:
T201603408.pdf Download
DOI
10.1007/s00439-016-1699-x
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Physiology (생리학교실) > 1. Journal Papers
Yonsei Authors
Kim, Dong Wook(김동욱) ORCID logo https://orcid.org/0000-0002-5025-1532
Park, Chul Yong(박철용) ORCID logo https://orcid.org/0000-0002-4467-9268
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/151941
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