CRISPR/Cas9 flexes its muscles: In vivo somatic gene editing for muscular dystrophy

Molecular Therapy

Volumn 24, Issue 3, 2016, Pages 414-416

  Van Den Driessche, Thierry ^a,b   Chuah, Marinee K ^a,b  

^a VRIJE UNIVERSITEIT BRUSSEL   (Belgium)

^b UNIVERSITY OF LEUVEN   (Belgium)

Author keywords

[No Author keywords available]

Indexed keywords

CRISPR ASSOCIATED PROTEIN; DYSTROPHIN; GUIDE RNA; VIRUS VECTOR; ADENO ASSOCIATED VIRUS VECTOR;

CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CRISPR CAS SYSTEM; DNA END JOINING REPAIR; DNA MODIFICATION; DNA TEMPLATE; DOUBLE STRANDED DNA BREAK; DUCHENNE MUSCULAR DYSTROPHY; ELECTROPORATION; EXON SKIPPING; GENE EXPRESSION REGULATION; GENE TARGETING; GENETIC VARIABILITY; HUMAN; IN VIVO GENE TRANSFER; INDEL MUTATION; MUSCLE STRENGTH; NONHUMAN; NOTE; PHENOTYPE; RNA VECTOR; SOMATIC GENE EDITING; SOMATIC GENE THERAPY; CAUSE OF DEATH; CRISPR-CAS9 SYSTEM; DISEASE SEVERITY; EXON; GENE EDITING; IN VIVO STUDY; INTRON; NONSENSE MUTATION; RESPIRATORY FAILURE; TISSUE DIFFERENTIATION; VIRAL GENE THERAPY; GENE THERAPY; GENETICS; MUSCULAR DYSTROPHY; RNA EDITING;

CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS; DYSTROPHIN; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHIES; RNA EDITING;

EID: 84960342156     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.29     Document Type: Note

References (29)

1. Ervasti, JM, Ohlendieck, K, Kahl, SD, Gaver, MG and Campbell, KP. (1990). Deficiency of a glycoprotein component of the dystrophin complex in dystrophic muscle. Nature 345: 315-319
2. Campbell, KP and Kahl, SD. (1989). Association of dystrophin and an integral membrane glycoprotein. Nature 338: 259-262
3. Xu, L, Park, KH, Zhao, L, Xu, J, El Refaey, M, Gao, Y, et al. (2016). CRISPR-mediated genome editing restores dystrophin expression and function in mdx mice. Mol Ther 24: 564-569
4. Long, C, Amoasii, L, Mireault, AA, McAnally, JR, Li, H, Sanchez-Ortiz, E, et al. (2016). Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351: 400-403
5. Nelson, CE, Hakim, CH, Ousterout, DG, Thakore, PI, Moreb, EA, Castellanos Rivera, RM, et al. (2016). In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351: 403-407
6. Tabebordbar, M, Zhu, K, Cheng, JK, Chew, WL, Widrick, JJ, Yan, WX, et al. (2016). In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351: 407-111
7. Loperfido, M, Jarmin, S, Dastidar, S, Di Matteo, M, Perini, I, Moore, M, et al. (2016). piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts. Nucleic Acids Res 44: 744-760
8. Gregorevic, P, Allen, JM, Minami, E, Blankinship, MJ, Haraguchi, M, Meuse, L, et al. (2006). rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 12: 787-789
9. Kawecka, K, Theodoulides, M, Hasoglu, Y, Jarmin, S, Kymalainen, H, Le-Heron, A, et al. (2015). Adenoassociated virus (AAV) mediated dystrophin gene transfer studies and exon skipping strategies for Duchenne muscular dystrophy (DMD). Curr Gene Ther 15: 395-415
10. van Deutekom, JC, Janson, AA, Ginjaar, IB, Frankhuizen, WS, Aartsma-Rus, A, Bremmer-Bout, M, et al. (2007). Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 357: 2677-2686
11. Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K, et al. (2011). Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 378: 595-605
12. Lu, QL, Cirak, S and Partridge, T. (2014). What can we learn from clinical trials of exon skipping for DMD?. Mol Ther Nucleic Acids 3: e152
13. Jinek, M, Chylinski, K, Fonfara, I, Hauer, M, Doudna, JA and Charpentier, E. (2012). A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337: 816-821
14. Mali, P, Yang, L, Esvelt, KM, Aach, J, Guell, M, DiCarlo, JE, et al. (2013). RNA-guided human genome engineering via Cas9. Science 339: 823-826
15. Cong, L, Ran, FA, Cox, D, Lin, S, Barretto, R, Habib, N, et al. (2013). Multiplex genome engineering using CRISPR/Cas systems. Science 339: 819-823
16. Cho, SW, Kim, S, Kim, JM and Kim, JS. (2013). Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol 31: 230-232
17. 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
18. Li, HL, Fujimoto, N, Sasakawa, N, Shirai, S, Ohkame, T, Sakuma, T, et al. (2015). Precise correction of the dystrophin gene in Duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9. Stem Cell Reports 4: 143-154
19. Iyombe-Engembe, JP, Ouellet, DL, Barbeau, X, Rousseau, J, Chapdelaine, P, Lagüe, P, et al. (2016). Efficient restoration of the dystrophin gene reading frame and protein structure in DMD myoblasts using the CinDel method. Mol Ther Nucleic Acids 5: e283
20. Maggio, I, Stefanucci, L, Janssen, JM, Liu, J, Chen, X, Mouly, V, et al. (2016). Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations. Nucleic Acids Res; e-pub ahead of print 13 January 2016
21. Long, C, McAnally, JR, Shelton, JM, Mireault, AA, Bassel-Duby, R and Olson, EN. (2014). Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science 345: 1184-1188
22. Gao, GP, Alvira, MR, Wang, L, Calcedo, R, Johnston, J and Wilson, JM. (2002). Novel adeno-Associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99: 11854-11859
23. Pacak, CA, Mah, CS, Thattaliyath, BD, Conlon, TJ, Lewis, MA, Cloutier, DE, et al. (2006). Recombinant adeno-Associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res 99: e3-9
24. VandenDriessche, T, Thorrez, L, Acosta-Sanchez, A, Petrus, I, Wang, L, Ma, L, et al. (2007). Efficacy and safety of adeno-Associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy. J Thromb Haemost 5: 16-24
25. Tsai, SQ, Zheng, Z, Nguyen, NT, Liebers, M, Topkar, VV, Thapar, V, et al. (2015). GUIDE-seq enables genome-wide profiling of off-Target cleavage by CRISPR-Cas nucleases. Nat Biotechnol 33: 187-197
26. Kim, D, Bae, S, Park, J, Kim, E, Kim, S, Yu, HR, et al. (2015). Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-Target effects in human cells. Nat Methods 12: 237-243
27. Kleinstiver, BP, Pattanayak, V, Prew, MS, Tsai, SQ, Nguyen, NT, Zheng, Z, et al. (2016). High-fidelity CRISPR-Cas9 nucleases with no detectable genomewide off-Target effects. Nature 529: 490-495
28. Wang, D, Mou, H, Li, S, Li, Y, Hough, S, Tran, K, et al. (2015). Adenovirus-mediated somatic genome editing of Pten by CRISPR/Cas9 in mouse liver in spite of Cas9-specific immune responses. Hum Gene Ther 26: 432-442
29. Rincon, MY, Sarcar, S, Danso-Abeam, D, Keyaerts, M, Matrai, J, Samara-Kuko, E, et al. (2015). Genomewide computational analysis reveals cardiomyocytespecific transcriptional cis-regulatory motifs that enable efficient cardiac gene therapy. Mol Ther 23: 43-52