Genome- and cell-based strategies in therapy of muscular dystrophies
- Авторлар: Lipinski M.1, Vassetzky Y.1,2, Bou Saada Y.1, Dib C.1
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Мекемелер:
- UMR 8126, CNRS, Université Paris-Sud
- Lomonosov Moscow State University
- Шығарылым: Том 81, № 7 (2016)
- Беттер: 678-690
- Бөлім: Review
- URL: https://journals.rcsi.science/0006-2979/article/view/150934
- DOI: https://doi.org/10.1134/S000629791607004X
- ID: 150934
Дәйексөз келтіру
Аннотация
Muscular dystrophies are a group of heterogeneous genetic disorders characterized by progressive loss of skeletal muscle mass. Depending on the muscular dystrophy, the muscle weakness varies in degree of severity. The majority of myopathies are due to genetic events leading to a loss of function of key genes involved in muscle function. Although there is until now no curative treatment to stop the progression of most myopathies, a significant number of experimental gene- and cell-based strategies and approaches have been and are being tested in vitro and in animal models, aiming to restore gene function. Genome editing using programmable endonucleases is a powerful tool for modifying target genome sequences and has been extensively used over the last decade to correct in vitro genetic defects of many single-gene diseases. By inducing double-strand breaks (DSBs), the engineered endonucleases specifically target chosen sequences. These DSBs are spontaneously repaired either by homologous recombination in the presence of a sequence template, or by nonhomologous-end joining error prone repair. In this review, we highlight recent developments and challenges for genome-editing based strategies that hold great promise for muscular dystrophies and regenerative medicine.
Негізгі сөздер
Авторлар туралы
M. Lipinski
UMR 8126, CNRS, Université Paris-Sud
Email: vassetzky@igr.fr
Франция, Villejuif, F-94805
Y. Vassetzky
UMR 8126, CNRS, Université Paris-Sud; Lomonosov Moscow State University
Хат алмасуға жауапты Автор.
Email: vassetzky@igr.fr
Франция, Villejuif, F-94805; Moscow, 119991
Y. Bou Saada
UMR 8126, CNRS, Université Paris-Sud
Email: vassetzky@igr.fr
Франция, Villejuif, F-94805
Carla Dib
UMR 8126, CNRS, Université Paris-Sud
Email: vassetzky@igr.fr
Франция, Villejuif, F-94805