Addressing challenges in the clinical applications associated with CRISPR/Cas9 technology and ethical questions to prevent its misuse

References

[1] BakondiB, LvW (2016) In vivo CRISPR/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of autosomal dominant retinitis pigmentosa. Mol Ther24(3):556–563

[2] BaltimoreD, BergP (2015) Biotechnology. A prudent path forward for genomic engineering and germline gene modification. Science348(6230):36–38

[3] BarrangouR, DoudnaJA (2016) Applications of CRISPR technologies in research and beyond. Nat Biotechnol34(9):933–941

[4] BikardD, EulerCW (2014) Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials. Nat Biotechnol32 (11):1146–1150

[5] BosleyKS, BotchanM (2015) CRISPR germline engineering—the community speaks. Nat Biotechnol33(5):478–486

[6] CoxDB, PlattRJ (2015) Therapeutic genome editing: prospects and challenges. Nat Med21(2):121–131

[7] DongC, QuL (2015) Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication. Antivir Res118:110–117

[8] FirthAL, MenonT (2015) Functional gene correction for cystic fibrosis in lung epithelial cells generated from patient iPSCs. Cell Rep12(9):1385–1390

[9] GomaaAA, KlumpeHE (2014) Programmable removal of bacterial strains by use of genome-targeting CRISPR-Cas systems. mBio5(1):e00913–e00928

[10] HouP, ChenS (2015) Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection. Sci Rep. 5:15577

[11] HsuPD, LanderES (2014) Development and applications of CRISPR-Cas9 for genome engineering. Cell157(6):1262–1278

[12] HuZ, YuL (2014) Disruption of HPV16-E7 by CRISPR/Cas system induces apoptosis and growth inhibition in HPV16 positive human cervical cancer cells. Biomed Res Int2014:612823

[13] HwangWY, FuY (2013) Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat Biotechnol31(3):227–229

[14] KangX, HeW (2016) Introducing precise genetic modifications into human 3PN embryos by CRISPR/Cas-mediated genome editing. J Assist Reprod Genet33(5):581–588

[15] LanphierE, UrnovF (2015) Don’t edit the human germ line. Nature519(7544):410–411

[16] LiC, GuanX (2015)Inhibition of HIV-1 infection of primary CD4+ T-cells by gene editing of CCR5 using adenovirusdelivered CRISPR/Cas9. J Gen Virol96(8):2381–2393

[17] LiangP, XuY (2015) CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell6(5):363–372

[18] LongC, McAnallyJR (2014) Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science345(6201):1184–1188

[19] MaH, Marti-GutierrezN (2017) Correction of a pathogenic gene mutation in human embryos. Nature548(7668):413–419

[20] MaederML, GersbachCA (2016) Genome-editing Technologies for Gene and Cell Therapy. Mol Ther24(3):430–446

[21] NelsonCE, HakimCH (2016) In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science351(6271):403–407

[22] OsbornMJ, GabrielR (2015) Fanconi anemia gene editing by the CRISPR/Cas9 system. Hum Gene Ther26(2):114–126

[23] PengJ, WangY (2015) Production of human albumin in pigs through CRISPR/Cas9-mediated knockin of human cDNA into swine albumin locus in the zygotes. Sci Rep5:16705

[24] RodriguezE (2016) Ethical issues in genome editing using Crispr/Cas9 system. J Clin Res Bioeth7(2):266

[25] SavulescuJ, PughJ (2015) The moral imperative to continue gene editing research on human embryos. Protein Cell6(7):476–479

[26] SchwankG, KooBK (2013) Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell Stem Cell13(6):653–658

[27] SvitashevS, YoungJK (2015) Targeted mutagenesis, precise gene editing, and site-specific gene insertion in maize using Cas9 and guide RNA. Plant Physiol169(2):931–945

[28] TangL, ZengY (2017) CRISPR/Cas9-mediated gene editing in human zygotes using Cas9 protein. Mol Genet Genomics292 (3):525–533

[29] TebasP, SteinD (2014) Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med370 (10):901–910

[30] TuZ, YangW (2017) Promoting Cas9 degradation reduces mosaic mutations in non-human primate embryos. Sci Rep7:42081

[31] VallettaS, DolatshadH (2015) ASXL1 mutation correction by CRISPR/Cas9 restores gene function in leukemia cells and increases survival in mouse xenografts. Oncotarget6 (42):44061–44071

[32] WangH, La RussaM (2016) CRISPR/Cas9 in genome editing and beyond. Annu Rev Biochem85:227–264

[33] WuYLD, WangY (2013) Correction of a genetic disease in mouse via use of CRISPR-Cas9. Cell Stem Cell13(6):659–662

[34] YangH, WangH (2013) One-step generation of mice carrying reporter and conditional alleles by CRISPR/Cas-mediated genome engineering. Cell154(6):1370–1379

[35] YangL, GuellM (2015) Genome-wide inactivation of porcine endogenous retroviruses (PERVs). Science350(6264):1101–1104

[36] YinH, XueW (2014) Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol32 (6):551–553

[37] YuL, WangX (2015) Disruption of human papillomavirus 16 E6 gene by clustered regularly interspaced short palindromic repeat/Cas system in human cervical cancer cells. OncoTargets Ther8:37–44

[38] ZetscheB, GootenbergJS (2015) Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system. Cell163 (3):759–771

RIGHTS & PERMISSIONS

2017 The Author(s) 2017. This article is an open access publication