Eliminate mitochondrial diseases by gene editing in germ-line cells and embryos

Protein and Cell

Volumn 6, Issue 7, 2015, Pages 472-475

  Wang, Si ^a   Yi, Fei ^b   Qu, Jing ^c  

^a INSTITUTE OF BIOPHYSICS   (China)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c INSTITUTE OF ZOOLOGY   (China)

Author keywords

[No Author keywords available]

Indexed keywords

CRISPR ASSOCIATED PROTEIN; CRISPR ASSOCIATED PROTEIN 9; GENOMIC DNA; HELPER DEPENDENT ADENOVIRAL VECTOR; HEMOGLOBIN BETA CHAIN; HEMOGLOBIN DELTA CHAIN; MITOCHONDRIAL DNA; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; UNCLASSIFIED DRUG; ZINC FINGER NUCLEASE;

ARTICLE; BETA THALASSEMIA; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; DISEASE COURSE; DISEASE TRANSMISSION; DISORDERS OF MITOCHONDRIAL FUNCTIONS; DNA TRANSFER; FERTILIZATION IN VITRO; GENE EDITING TECHNOLOGY; GENE TARGETING; GENETIC PROCEDURES; GERM CELL; HAPLOTYPE; HUMAN; MITOCHONDRIAL RESPIRATION; NONHUMAN; NUCLEASE BASED GENOME EDITING; OOCYTE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; SOMATIC CELL; ZYGOTE; ANIMAL; GENETICS; GENOME; MAMMALIAN EMBRYO; METABOLISM; MITOCHONDRIAL DISEASES; RNA EDITING;

ANIMALS; DNA, MITOCHONDRIAL; EMBRYO, MAMMALIAN; GENOME; GERM CELLS; HUMANS; MITOCHONDRIAL DISEASES; RNA EDITING;

EID: 84943361624     PISSN: 1674800X     EISSN: 16748018     Source Type: Journal    
DOI: 10.1007/s13238-015-0177-x     Document Type: Article

References (24)

1. Hai T, Teng F, Guo R, Li W, Zhou Q (2014) One-step generation of knockout pigs by zygote injection of CRISPR/Cas system. Cell Res 24:372–375
2. Hu W, Kaminski R, Yang F, Zhang Y, Cosentino L, Li F, Luo B, Alvarez-Carbonell D, Garcia-Mesa Y, Karn J et al (2014) RNAdirected gene editing specifically eradicates latent and prevents new HIV-1 infection. Proc Natl Acad Sci U S A 111:11461–11466
3. Li M, Suzuki K, Qu J, Saini P, Dubova I, Yi F, Lee J, Sancho-Martinez I, Liu GH, Izpisua Belmonte JC (2011) Efficient correction of hemoglobinopathy-causing mutations by homologous recombination in integration-free patient iPSCs. Cell Res 21:1740–1744
4. Liang P, Xu Y, Zhang X, Ding C, Huang R, Zhang Z, Lv J, Xie X, Chen Y, Li Y et al (2015) CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell 6:363–372
5. Liu GH, Suzuki K, Qu J, Sancho-Martinez I, Yi F, Li M, Kumar S, Nivet E, Kim J, Soligalla RD et al (2011) Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs. Cell Stem Cell 8:688–694
6. Liu GH, Qu J, Suzuki K, Nivet E, Li M, Montserrat N, Yi F, Xu X, Ruiz S, Zhang W et al (2012) Progressive degeneration of human neural stem cells caused by pathogenic LRRK2. Nature 491:603–607
7. Liu GH, Suzuki K, Li M, Qu J, Montserrat N, Tarantino C, Gu Y, Yi F, Xu X, Zhang W et al (2014) Modelling Fanconi anemia pathogenesis and therapeutics using integration-free patient-derived iPSCs. Nat Commun 5:4330
8. Mandal PK, Ferreira LM, Collins R, Meissner TB, Boutwell CL, Friesen M, Vrbanac V, Garrison BS, Stortchevoi A, Bryder D et al (2014) Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9. Cell Stem Cell 15:643–652
9. Niu Y, Shen B, Cui Y, Chen Y, Wang J, Wang L, Kang Y, Zhao X, Si W, Li W et al (2014) Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos. Cell 156:836–843
10. Ousterout DG, Kabadi AM, Thakore PI, Majoros WH, Reddy TE, Gersbach CA (2015) Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy. Nat Commun 6:6244
11. Paull D, Emmanuele V, Weiss KA, Treff N, Stewart L, Hua H, Zimmer M, Kahler DJ, Goland RS, Noggle SA et al (2013) Nuclear genome transfer in human oocytes eliminates mitochondrial DNA variants. Nature 493:632–637
12. Reddy P, Ocampo A, Suzuki K, Luo J, Bacman SR, Williams SL, Sugawara A, Okamura D, Tsunekawa Y, Wu J et al (2015) Selective elimination of mitochondrial mutations in the germline by genome editing. Cell 161:459–469
13. Smith C, Gore A, Yan W, Abalde-Atristain L, Li Z, He C, Wang Y, Brodsky RA, Zhang K, Cheng L et al (2014) Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs. Cell Stem Cell 15:12–13
14. Suzuki K, Yu C, Qu J, Li M, Yao X, Yuan T, Goebl A, Tang S, Ren R, Aizawa E et al (2014) Targeted gene correction minimally impacts whole-genome mutational load in human-disease-specific induced pluripotent stem cell clones. Cell Stem Cell 15:31–36
15. Tachibana M, Amato P, Sparman M, Woodward J, Sanchis DM, Ma H, Gutierrez NM, Tippner-Hedges R, Kang E, Lee HS et al (2013) Towards germline gene therapy of inherited mitochondrial diseases. Nature 493:627–631
16. Taylor RW, Turnbull DM (2005) Mitochondrial DNA mutations in human disease. Nat Rev Genet 6:389–402
17. Veres A, Gosis BS, Ding Q, Collins R, Ragavendran A, Brand H, Erdin S, Cowan CA, Talkowski ME, Musunuru K (2014) Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by wholegenome sequencing. Cell Stem Cell 15:27–30
18. Wang H, Yang H, Shivalila CS, Dawlaty MM, Cheng AW, Zhang F, Jaenisch R (2013) One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 153:910–918
19. Wang T, Sha H, Ji D, Zhang HL, Chen D, Cao Y, Zhu J (2014) Polar body genome transfer for preventing the transmission of inherited mitochondrial diseases. Cell 157:1591–1604
20. Xie F, Ye L, Chang JC, Beyer AI, Wang J, Muench MO, Kan YW (2014) Seamless gene correction of beta-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac. Genome Res 24:1526–1533
21. Xu X, Qu J, Suzuki K, Li M, Zhang W, Liu GH, Izpisua Belmonte JC (2012) Reprogramming based gene therapy for inherited red blood cell disorders. Cell Res 22:941–944
22. Xu X, Duan S, Yi F, Ocampo A, Liu GH, Izpisua Belmonte JC (2013) Mitochondrial regulation in pluripotent stem cells. Cell Metab 18:325–332
23. Yang H, Wang H, Shivalila CS, Cheng AW, Shi L, Jaenisch R (2013) One-step generation of mice carrying reporter and conditional alleles by CRISPR/Cas-mediated genome engineering. Cell 154:1370–1379
24. Yang L, Grishin D, Wang G, Aach J, Zhang CZ, Chari R, Homsy J, Cai X, Zhao Y, Fan JB et al (2014) Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells. Nat Commun 5:5507