Applications of Gene Editing in Mitochondrial Diseases

被引:0
|
作者
Chang, Liying [1 ,2 ,3 ]
Ling, Xinyu [1 ,2 ,3 ]
Chen, Heqi [1 ,2 ,3 ]
Wang, Xue [1 ,2 ,3 ]
Liu, Tao [1 ,2 ,3 ]
机构
[1] Sch Pharmaceut Sci, State Key Lab Nat & Biomimet Drugs, Beijing 100191, Peoples R China
[2] Peking Univ, Chem Biol Ctr, Beijing 100191, Peoples R China
[3] Peking Univ, Sch Pharmaceut Sci, Dept Mol & Cellular Pharmacol, Beijing 100191, Peoples R China
来源
基金
美国国家科学基金会;
关键词
Mitochondrial diseases; Mitochondrial heteroplasmy; Gene editing; HEREDITARY OPTIC NEUROPATHY; MTDNA HETEROPLASMY; MUTANT MTDNA; SELECTIVE ELIMINATION; ARGONAUTE PROTEINS; DNA HETEROPLASMY; MITO-PORTER; DELIVERY; RNA; THERAPY;
D O I
10.7503/cjcu20220363
中图分类号
O6 [化学];
学科分类号
0703 ;
摘要
Mitochondria, as energy factories of cells, play an important role in maintaining cell energy metabolism and human life activities. The mutations of mitochondrial genome have led to a series of mitochondrial diseases, which seriously threaten human life and health. The development of gene editing methods targeting mitochondria is of great significance for the treatment of mitochondrial diseases. In recent years, a series of gene editing methods inclu- ding restriction nuclease, zinc finger nuclease, transcriptional activator like effector nuclease, clustered regularly interspaced short palindromic repeats(CRISPR) and mitochondrial base editor have been developed. This article reviewed the recent progresses, deficiencies and prospects of the applications of gene editing tools for mitochondria gene editing. We hope it could provide reference for the development of mitochondrial disease treatment.
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页数:11
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共 104 条
  • [1] Alexeyev MF, 2008, GENE THER, V15, P516, DOI [10.1038/gt.2008.11, 10.1038/sj.gt.2008.11]
  • [2] SEQUENCE AND ORGANIZATION OF THE HUMAN MITOCHONDRIAL GENOME
    ANDERSON, S
    BANKIER, AT
    BARRELL, BG
    DEBRUIJN, MHL
    COULSON, AR
    DROUIN, J
    EPERON, IC
    NIERLICH, DP
    ROE, BA
    SANGER, F
    SCHREIER, PH
    SMITH, AJH
    STADEN, R
    YOUNG, IG
    [J]. NATURE, 1981, 290 (5806) : 457 - 465
  • [3] Mitochondrial import, health and mtDNA copy number variability seen when using type II and type V CRISPR effectors
    Anton, Zurine
    Mullally, Grace
    Ford, Holly C.
    van der Kamp, Marc W.
    Szczelkun, Mark D.
    Lane, Jon D.
    [J]. JOURNAL OF CELL SCIENCE, 2020, 133 (18)
  • [4] Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors
    Anzalone, Andrew V.
    Koblan, Luke W.
    Liu, David R.
    [J]. NATURE BIOTECHNOLOGY, 2020, 38 (07) : 824 - 844
  • [5] Engineering of large numbers of highly specific homing endonucleases that induce recombination on novel DNA targets
    Arnould, S
    Chames, P
    Perez, C
    Lacroix, E
    Duclert, A
    Epinat, JC
    Stricher, F
    Petit, AS
    Patin, A
    Guillier, S
    Rolland, S
    Prieto, J
    Blanco, FJ
    Bravo, J
    Montoya, G
    Serrano, L
    Duchateau, P
    Pâques, F
    [J]. JOURNAL OF MOLECULAR BIOLOGY, 2006, 355 (03) : 443 - 458
  • [6] Modulating mtDNA heteroplasmy by mitochondria-targeted restriction endonucleases in a 'differential multiple cleavage-site' model
    Bacman, S. R.
    Williams, S. L.
    Hernandez, D.
    Moraes, C. T.
    [J]. GENE THERAPY, 2007, 14 (18) : 1309 - 1318
  • [7] Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease
    Bacman, S. R.
    Williams, S. L.
    Duan, D.
    Moraes, C. T.
    [J]. GENE THERAPY, 2012, 19 (11) : 1101 - 1106
  • [8] Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease
    Bacman, S. R.
    Williams, S. L.
    Garcia, S.
    Moraes, C. T.
    [J]. GENE THERAPY, 2010, 17 (06) : 713 - 720
  • [9] MitoTALEN reduces mutant mtDNA load and restores tRNAAla levels in a mouse model of heteroplasmic mtDNA mutation
    Bacman, Sandra R.
    Kauppila, Johanna H. K.
    Pereira, Claudia, V
    Nissanka, Nadee
    Miranda, Maria
    Pinto, Milena
    Williams, Sion L.
    Larsson, Nils-Goeran
    Stewart, James B.
    Moraes, Carlos T.
    [J]. NATURE MEDICINE, 2018, 24 (11) : 1696 - +
  • [10] Specific elimination of mutant mitochondrial genomes in patient-derived cells by mitoTALENs
    Bacman, Sandra R.
    Williams, Sion L.
    Pinto, Milena
    Peralta, Susana
    Moraes, Carlos T.
    [J]. NATURE MEDICINE, 2013, 19 (09) : 1111 - 1113