Cancer gene therapy: innovations in therapeutic delivery of CRISPR-Cas9

Gene therapy has great potential for use in safe and effective cancer treatments, as it can target oncogenic pathways at a molecular level. The recent development of a genome editing system using clustered regularly interspersed palindromic repeats (CRISPR) has made gene therapy for human diseases m...

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Published inDrug discovery today. Disease models Vol. 21; pp. 17 - 21
Main Authors Lindsay-Mosher, Nicole, Su, Cathy
Format Journal Article
LanguageEnglish
Published Elsevier Ltd 2016
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Abstract Gene therapy has great potential for use in safe and effective cancer treatments, as it can target oncogenic pathways at a molecular level. The recent development of a genome editing system using clustered regularly interspersed palindromic repeats (CRISPR) has made gene therapy for human diseases much more feasible. To realize the full potential of CRISPR and CRISPR-associated systems (Cas) for cancer treatment, however, this gene editing system must be efficiently and safely delivered to cancer cells in vivo. Here we review innovations made in the design of both viral and non-viral vectors to accommodate CRISPR-Cas systems, addressing the challenges of size constraints, immunogenicity, and specificity.
AbstractList Gene therapy has great potential for use in safe and effective cancer treatments, as it can target oncogenic pathways at a molecular level. The recent development of a genome editing system using clustered regularly interspersed palindromic repeats (CRISPR) has made gene therapy for human diseases much more feasible. To realize the full potential of CRISPR and CRISPR-associated systems (Cas) for cancer treatment, however, this gene editing system must be efficiently and safely delivered to cancer cells in vivo. Here we review innovations made in the design of both viral and non-viral vectors to accommodate CRISPR-Cas systems, addressing the challenges of size constraints, immunogenicity, and specificity.
Author Su, Cathy
Lindsay-Mosher, Nicole
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  givenname: Cathy
  surname: Su
  fullname: Su, Cathy
  organization: Department of Physics, University of Toronto, Toronto, Canada
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Cites_doi 10.1038/nature.2016.20988
10.1002/jgm.2698
10.1038/srep10777
10.1038/nrg1066
10.2174/156652311795684740
10.1002/anie.201506030
10.1038/nrg3763
10.1101/gr.171322.113
10.1038/nbt.3471
10.1038/nmeth.3993
10.1002/adhm.201400235
10.1002/anie.201610209
10.1039/C6BM00441E
10.1128/JVI.00649-08
10.1038/ncomms13056
10.1172/JCI35700
10.1073/pnas.1520244113
10.1038/mtm.2016.57
10.2174/1566523213666131125095046
10.1038/nbt.3659
10.1038/gt.2009.148
10.1038/ncomms12868
10.1038/srep05105
10.12703/P6-3
10.2174/138161210789941847
10.1016/j.gene.2013.03.137
10.1038/nbt.3081
10.1101/gr.162339.113
10.1089/hum.2009.182
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References Wirth (bib0025) 2013; 525
Miller (bib0150) 2017; 56
Auman (bib0090) 2010; 12
Wold, Toth (bib0100) 2013; 13
Boutin (bib0110) 2010; 21
Urruticoechea (bib0010) 2010; 16
Keles (bib0175) 2016; 4
Segovia (bib0190) 2015; 4
(accessed May 1, 2017).
Gene Therapy Clinical Trials Worldwide n.d.
Zuris (bib0160) 2015; 33
Canadian Cancer Society. Canadian Cancer Statistics 2015. Toronto: 2015.
Yin (bib0060) 2016; 34
Yin (bib0135) 2014; 15
Ma (bib0120) 2016; 7
Park (bib0130) 2016; 3
Nayak, Herzog (bib0140) 2010; 17
Yi (bib0095) 2011; 11
Ginn (bib0065) 2013; 15
Wu (bib0080) 2014; 2
Sibbald (bib0015) 2001; 164
Fine (bib0115) 2015; 5
Kim (bib0155) 2014; 24
Maggio (bib0085) 2014; 4
Gilam (bib0195) 2016; 7
Reardon (bib0045) 2016
ClinicalTrials.gov n.d.
Thomas (bib0070) 2003; 4
Penaud-Budloo (bib0105) 2008; 82
Hacein-Bey-Abina (bib0020) 2008; 118
Lin (bib0185) 2016
Cho (bib0075) 2014; 24
Wang (bib0165) 2016; 113
Sun (bib0170) 2015; 54
Wilkinson, Wiedenheft (bib0055) 2014; 6
Chew (bib0125) 2016
Wang (bib0145) 2016
Barrangou, Doudna (bib0035) 2016; 34
Cyranoski (bib0040) 2016; 539
Wang, Burdick (bib0180) 2016
10.1016/j.ddmod.2017.02.009_bib0030
Barrangou (10.1016/j.ddmod.2017.02.009_bib0035) 2016; 34
Wang (10.1016/j.ddmod.2017.02.009_bib0180) 2016
Wilkinson (10.1016/j.ddmod.2017.02.009_bib0055) 2014; 6
Urruticoechea (10.1016/j.ddmod.2017.02.009_bib0010) 2010; 16
Ma (10.1016/j.ddmod.2017.02.009_bib0120) 2016; 7
10.1016/j.ddmod.2017.02.009_bib0050
Park (10.1016/j.ddmod.2017.02.009_bib0130) 2016; 3
Lin (10.1016/j.ddmod.2017.02.009_bib0185) 2016
Kim (10.1016/j.ddmod.2017.02.009_bib0155) 2014; 24
Yin (10.1016/j.ddmod.2017.02.009_bib0135) 2014; 15
Wang (10.1016/j.ddmod.2017.02.009_bib0145) 2016
Wold (10.1016/j.ddmod.2017.02.009_bib0100) 2013; 13
Keles (10.1016/j.ddmod.2017.02.009_bib0175) 2016; 4
Gilam (10.1016/j.ddmod.2017.02.009_bib0195) 2016; 7
Segovia (10.1016/j.ddmod.2017.02.009_bib0190) 2015; 4
Wu (10.1016/j.ddmod.2017.02.009_bib0080) 2014; 2
Yin (10.1016/j.ddmod.2017.02.009_bib0060) 2016; 34
Chew (10.1016/j.ddmod.2017.02.009_bib0125) 2016
Fine (10.1016/j.ddmod.2017.02.009_bib0115) 2015; 5
Miller (10.1016/j.ddmod.2017.02.009_bib0150) 2017; 56
Auman (10.1016/j.ddmod.2017.02.009_bib0090) 2010; 12
Hacein-Bey-Abina (10.1016/j.ddmod.2017.02.009_bib0020) 2008; 118
Cyranoski (10.1016/j.ddmod.2017.02.009_bib0040) 2016; 539
Nayak (10.1016/j.ddmod.2017.02.009_bib0140) 2010; 17
Maggio (10.1016/j.ddmod.2017.02.009_bib0085) 2014; 4
Wang (10.1016/j.ddmod.2017.02.009_bib0165) 2016; 113
10.1016/j.ddmod.2017.02.009_bib0005
Sun (10.1016/j.ddmod.2017.02.009_bib0170) 2015; 54
Wirth (10.1016/j.ddmod.2017.02.009_bib0025) 2013; 525
Thomas (10.1016/j.ddmod.2017.02.009_bib0070) 2003; 4
Sibbald (10.1016/j.ddmod.2017.02.009_bib0015) 2001; 164
Cho (10.1016/j.ddmod.2017.02.009_bib0075) 2014; 24
Zuris (10.1016/j.ddmod.2017.02.009_bib0160) 2015; 33
Ginn (10.1016/j.ddmod.2017.02.009_bib0065) 2013; 15
Reardon (10.1016/j.ddmod.2017.02.009_bib0045) 2016
Penaud-Budloo (10.1016/j.ddmod.2017.02.009_bib0105) 2008; 82
Boutin (10.1016/j.ddmod.2017.02.009_bib0110) 2010; 21
Yi (10.1016/j.ddmod.2017.02.009_bib0095) 2011; 11
References_xml – volume: 164
  start-page: 1612
  year: 2001
  ident: bib0015
  article-title: Death but one unintended consequence of gene-therapy trial
  publication-title: CMAJ
  contributor:
    fullname: Sibbald
– volume: 5
  start-page: 10777
  year: 2015
  ident: bib0115
  article-title: Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes
  publication-title: Sci Rep
  contributor:
    fullname: Fine
– volume: 34
  start-page: 328
  year: 2016
  end-page: 333
  ident: bib0060
  article-title: Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
  publication-title: Nat Biotechnol
  contributor:
    fullname: Yin
– volume: 113
  start-page: 2868
  year: 2016
  end-page: 2873
  ident: bib0165
  article-title: Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles
  publication-title: Proc Natl Acad Sci U S A
  contributor:
    fullname: Wang
– volume: 4
  start-page: 346
  year: 2003
  end-page: 358
  ident: bib0070
  article-title: Progress and problems with the use of viral vectors for gene therapy
  publication-title: Nat Rev Genet
  contributor:
    fullname: Thomas
– volume: 118
  start-page: 3132
  year: 2008
  end-page: 3142
  ident: bib0020
  article-title: Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
  publication-title: J Clin Invest
  contributor:
    fullname: Hacein-Bey-Abina
– volume: 24
  start-page: 132
  year: 2014
  end-page: 141
  ident: bib0075
  article-title: Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
  publication-title: Genome Res
  contributor:
    fullname: Cho
– volume: 15
  start-page: 541
  year: 2014
  end-page: 555
  ident: bib0135
  article-title: Non-viral vectors for gene-based therapy
  publication-title: Nat Rev Genet
  contributor:
    fullname: Yin
– volume: 82
  start-page: 7875
  year: 2008
  end-page: 7885
  ident: bib0105
  article-title: Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle
  publication-title: J Virol
  contributor:
    fullname: Penaud-Budloo
– volume: 15
  start-page: 65
  year: 2013
  end-page: 77
  ident: bib0065
  article-title: Gene therapy clinical trials worldwide to 2012—an update
  publication-title: J Gene Med
  contributor:
    fullname: Ginn
– volume: 7
  start-page: 12868
  year: 2016
  ident: bib0195
  article-title: Local microRNA delivery targets Palladin and prevents metastatic breast cancer
  publication-title: Nat Commun
  contributor:
    fullname: Gilam
– volume: 16
  start-page: 3
  year: 2010
  end-page: 10
  ident: bib0010
  article-title: Recent advances in cancer therapy: an overview
  publication-title: Curr Pharm Des
  contributor:
    fullname: Urruticoechea
– volume: 7
  start-page: 13056
  year: 2016
  ident: bib0120
  article-title: Integration and exchange of split dCas9 domains for transcriptional controls in mammalian cells
  publication-title: Nat Commun
  contributor:
    fullname: Ma
– year: 2016
  ident: bib0045
  article-title: First CRISPR clinical trial gets green light from US panel
  publication-title: Nature
  contributor:
    fullname: Reardon
– volume: 17
  start-page: 295
  year: 2010
  end-page: 304
  ident: bib0140
  article-title: Progress and prospects: immune responses to viral vectors
  publication-title: Gene Ther
  contributor:
    fullname: Herzog
– volume: 56
  start-page: 1059
  year: 2017
  end-page: 1063
  ident: bib0150
  article-title: Non-viral CRISPR/Cas gene editing in vitro and in vivo enabled by synthetic nanoparticle co-delivery of Cas9 mRNA and sgRNA
  publication-title: Angew Chem Int Ed
  contributor:
    fullname: Miller
– year: 2016
  ident: bib0185
  article-title: Injectable supramolecular hydrogel formed from α-cyclodextrin and PEGylated arginine-functionalized poly(
  publication-title: Acta Biomater
  contributor:
    fullname: Lin
– volume: 6
  start-page: 3
  year: 2014
  ident: bib0055
  article-title: A CRISPR method for genome engineering
  publication-title: F1000Prime Rep
  contributor:
    fullname: Wiedenheft
– volume: 4
  start-page: 271
  year: 2015
  end-page: 280
  ident: bib0190
  article-title: Hydrogel doped with nanoparticles for local sustained release of siRNA in breast cancer
  publication-title: Adv Healthc Mater
  contributor:
    fullname: Segovia
– volume: 4
  start-page: 5105
  year: 2014
  ident: bib0085
  article-title: Adenoviral vector delivery of RNA-guided CRISPR/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells
  publication-title: Sci Rep
  contributor:
    fullname: Maggio
– volume: 21
  start-page: 704
  year: 2010
  end-page: 712
  ident: bib0110
  article-title: Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors
  publication-title: Hum Gene Ther
  contributor:
    fullname: Boutin
– start-page: 1601041
  year: 2016
  ident: bib0180
  article-title: Engineered hydrogels for local and sustained delivery of RNA-interference therapies
  publication-title: Adv Healthc Mater
  contributor:
    fullname: Burdick
– volume: 4
  start-page: 1291
  year: 2016
  end-page: 1309
  ident: bib0175
  article-title: Recent progress in nanomaterials for gene delivery applications
  publication-title: Biomater Sci
  contributor:
    fullname: Keles
– year: 2016
  ident: bib0125
  article-title: A multifunctional AAV–CRISPR–Cas9 and its host response
  publication-title: Nat Methods
  contributor:
    fullname: Chew
– volume: 11
  start-page: 218
  year: 2011
  end-page: 228
  ident: bib0095
  article-title: Current advances in retroviral gene therapy
  publication-title: Curr Gene Ther
  contributor:
    fullname: Yi
– volume: 34
  start-page: 933
  year: 2016
  end-page: 941
  ident: bib0035
  article-title: Applications of CRISPR technologies in research and beyond
  publication-title: Nat Biotechnol
  contributor:
    fullname: Doudna
– volume: 3
  start-page: 16057
  year: 2016
  ident: bib0130
  article-title: Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing
  publication-title: Mol Ther Methods Clin Dev
  contributor:
    fullname: Park
– volume: 24
  start-page: 1012
  year: 2014
  end-page: 1019
  ident: bib0155
  article-title: Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
  publication-title: Genome Res
  contributor:
    fullname: Kim
– volume: 54
  start-page: 12029
  year: 2015
  end-page: 12033
  ident: bib0170
  article-title: Self-assembled DNA nanoclews for the efficient delivery of CRISPR-Cas9 for genome editing
  publication-title: Angew Chem Int Ed Engl
  contributor:
    fullname: Sun
– volume: 2
  start-page: 59
  year: 2014
  end-page: 70
  ident: bib0080
  article-title: Target specificity of the CRISPR-Cas9 system
  publication-title: Quant Biol (Beijing, China)
  contributor:
    fullname: Wu
– volume: 12
  start-page: 637
  year: 2010
  end-page: 638
  ident: bib0090
  article-title: Gene therapy: have the risks associated with viral vectors been solved?
  publication-title: Curr Opin Mol Ther
  contributor:
    fullname: Auman
– volume: 13
  start-page: 421
  year: 2013
  end-page: 433
  ident: bib0100
  article-title: Adenovirus vectors for gene therapy, vaccination and cancer gene therapy
  publication-title: Curr Gene Ther
  contributor:
    fullname: Toth
– volume: 33
  start-page: 73
  year: 2015
  end-page: 80
  ident: bib0160
  article-title: Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
  publication-title: Nat Biotechnol
  contributor:
    fullname: Zuris
– start-page: 17
  year: 2016
  ident: bib0145
  article-title: In vivo delivery systems for therapeutic genome editing
  publication-title: Int J Mol Sci
  contributor:
    fullname: Wang
– volume: 525
  start-page: 162
  year: 2013
  end-page: 169
  ident: bib0025
  article-title: History of gene therapy
  publication-title: Gene
  contributor:
    fullname: Wirth
– volume: 539
  year: 2016
  ident: bib0040
  article-title: CRISPR gene-editing tested in a person for the first time
  publication-title: Nature
  contributor:
    fullname: Cyranoski
– volume: 539
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0040
  article-title: CRISPR gene-editing tested in a person for the first time
  publication-title: Nature
  doi: 10.1038/nature.2016.20988
  contributor:
    fullname: Cyranoski
– year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0045
  article-title: First CRISPR clinical trial gets green light from US panel
  publication-title: Nature
  contributor:
    fullname: Reardon
– volume: 15
  start-page: 65
  year: 2013
  ident: 10.1016/j.ddmod.2017.02.009_bib0065
  article-title: Gene therapy clinical trials worldwide to 2012—an update
  publication-title: J Gene Med
  doi: 10.1002/jgm.2698
  contributor:
    fullname: Ginn
– start-page: 17
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0145
  article-title: In vivo delivery systems for therapeutic genome editing
  publication-title: Int J Mol Sci
  contributor:
    fullname: Wang
– volume: 5
  start-page: 10777
  year: 2015
  ident: 10.1016/j.ddmod.2017.02.009_bib0115
  article-title: Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes
  publication-title: Sci Rep
  doi: 10.1038/srep10777
  contributor:
    fullname: Fine
– volume: 2
  start-page: 59
  year: 2014
  ident: 10.1016/j.ddmod.2017.02.009_bib0080
  article-title: Target specificity of the CRISPR-Cas9 system
  publication-title: Quant Biol (Beijing, China)
  contributor:
    fullname: Wu
– volume: 4
  start-page: 346
  year: 2003
  ident: 10.1016/j.ddmod.2017.02.009_bib0070
  article-title: Progress and problems with the use of viral vectors for gene therapy
  publication-title: Nat Rev Genet
  doi: 10.1038/nrg1066
  contributor:
    fullname: Thomas
– volume: 11
  start-page: 218
  year: 2011
  ident: 10.1016/j.ddmod.2017.02.009_bib0095
  article-title: Current advances in retroviral gene therapy
  publication-title: Curr Gene Ther
  doi: 10.2174/156652311795684740
  contributor:
    fullname: Yi
– ident: 10.1016/j.ddmod.2017.02.009_bib0050
– volume: 54
  start-page: 12029
  year: 2015
  ident: 10.1016/j.ddmod.2017.02.009_bib0170
  article-title: Self-assembled DNA nanoclews for the efficient delivery of CRISPR-Cas9 for genome editing
  publication-title: Angew Chem Int Ed Engl
  doi: 10.1002/anie.201506030
  contributor:
    fullname: Sun
– volume: 15
  start-page: 541
  year: 2014
  ident: 10.1016/j.ddmod.2017.02.009_bib0135
  article-title: Non-viral vectors for gene-based therapy
  publication-title: Nat Rev Genet
  doi: 10.1038/nrg3763
  contributor:
    fullname: Yin
– ident: 10.1016/j.ddmod.2017.02.009_bib0005
– volume: 12
  start-page: 637
  year: 2010
  ident: 10.1016/j.ddmod.2017.02.009_bib0090
  article-title: Gene therapy: have the risks associated with viral vectors been solved?
  publication-title: Curr Opin Mol Ther
  contributor:
    fullname: Auman
– volume: 24
  start-page: 1012
  year: 2014
  ident: 10.1016/j.ddmod.2017.02.009_bib0155
  article-title: Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
  publication-title: Genome Res
  doi: 10.1101/gr.171322.113
  contributor:
    fullname: Kim
– start-page: 1601041
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0180
  article-title: Engineered hydrogels for local and sustained delivery of RNA-interference therapies
  publication-title: Adv Healthc Mater
  contributor:
    fullname: Wang
– volume: 34
  start-page: 328
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0060
  article-title: Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
  publication-title: Nat Biotechnol
  doi: 10.1038/nbt.3471
  contributor:
    fullname: Yin
– year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0125
  article-title: A multifunctional AAV–CRISPR–Cas9 and its host response
  publication-title: Nat Methods
  doi: 10.1038/nmeth.3993
  contributor:
    fullname: Chew
– volume: 4
  start-page: 271
  year: 2015
  ident: 10.1016/j.ddmod.2017.02.009_bib0190
  article-title: Hydrogel doped with nanoparticles for local sustained release of siRNA in breast cancer
  publication-title: Adv Healthc Mater
  doi: 10.1002/adhm.201400235
  contributor:
    fullname: Segovia
– volume: 56
  start-page: 1059
  year: 2017
  ident: 10.1016/j.ddmod.2017.02.009_bib0150
  article-title: Non-viral CRISPR/Cas gene editing in vitro and in vivo enabled by synthetic nanoparticle co-delivery of Cas9 mRNA and sgRNA
  publication-title: Angew Chem Int Ed
  doi: 10.1002/anie.201610209
  contributor:
    fullname: Miller
– volume: 4
  start-page: 1291
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0175
  article-title: Recent progress in nanomaterials for gene delivery applications
  publication-title: Biomater Sci
  doi: 10.1039/C6BM00441E
  contributor:
    fullname: Keles
– year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0185
  article-title: Injectable supramolecular hydrogel formed from α-cyclodextrin and PEGylated arginine-functionalized poly(l-lysine) dendron for sustained MMP-9 shRNA plasmid delivery
  publication-title: Acta Biomater
  contributor:
    fullname: Lin
– volume: 82
  start-page: 7875
  year: 2008
  ident: 10.1016/j.ddmod.2017.02.009_bib0105
  article-title: Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle
  publication-title: J Virol
  doi: 10.1128/JVI.00649-08
  contributor:
    fullname: Penaud-Budloo
– volume: 7
  start-page: 13056
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0120
  article-title: Integration and exchange of split dCas9 domains for transcriptional controls in mammalian cells
  publication-title: Nat Commun
  doi: 10.1038/ncomms13056
  contributor:
    fullname: Ma
– volume: 164
  start-page: 1612
  year: 2001
  ident: 10.1016/j.ddmod.2017.02.009_bib0015
  article-title: Death but one unintended consequence of gene-therapy trial
  publication-title: CMAJ
  contributor:
    fullname: Sibbald
– volume: 118
  start-page: 3132
  year: 2008
  ident: 10.1016/j.ddmod.2017.02.009_bib0020
  article-title: Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
  publication-title: J Clin Invest
  doi: 10.1172/JCI35700
  contributor:
    fullname: Hacein-Bey-Abina
– volume: 113
  start-page: 2868
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0165
  article-title: Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles
  publication-title: Proc Natl Acad Sci U S A
  doi: 10.1073/pnas.1520244113
  contributor:
    fullname: Wang
– volume: 3
  start-page: 16057
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0130
  article-title: Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing
  publication-title: Mol Ther Methods Clin Dev
  doi: 10.1038/mtm.2016.57
  contributor:
    fullname: Park
– volume: 13
  start-page: 421
  year: 2013
  ident: 10.1016/j.ddmod.2017.02.009_bib0100
  article-title: Adenovirus vectors for gene therapy, vaccination and cancer gene therapy
  publication-title: Curr Gene Ther
  doi: 10.2174/1566523213666131125095046
  contributor:
    fullname: Wold
– volume: 34
  start-page: 933
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0035
  article-title: Applications of CRISPR technologies in research and beyond
  publication-title: Nat Biotechnol
  doi: 10.1038/nbt.3659
  contributor:
    fullname: Barrangou
– volume: 17
  start-page: 295
  year: 2010
  ident: 10.1016/j.ddmod.2017.02.009_bib0140
  article-title: Progress and prospects: immune responses to viral vectors
  publication-title: Gene Ther
  doi: 10.1038/gt.2009.148
  contributor:
    fullname: Nayak
– ident: 10.1016/j.ddmod.2017.02.009_bib0030
– volume: 7
  start-page: 12868
  year: 2016
  ident: 10.1016/j.ddmod.2017.02.009_bib0195
  article-title: Local microRNA delivery targets Palladin and prevents metastatic breast cancer
  publication-title: Nat Commun
  doi: 10.1038/ncomms12868
  contributor:
    fullname: Gilam
– volume: 4
  start-page: 5105
  year: 2014
  ident: 10.1016/j.ddmod.2017.02.009_bib0085
  article-title: Adenoviral vector delivery of RNA-guided CRISPR/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells
  publication-title: Sci Rep
  doi: 10.1038/srep05105
  contributor:
    fullname: Maggio
– volume: 6
  start-page: 3
  year: 2014
  ident: 10.1016/j.ddmod.2017.02.009_bib0055
  article-title: A CRISPR method for genome engineering
  publication-title: F1000Prime Rep
  doi: 10.12703/P6-3
  contributor:
    fullname: Wilkinson
– volume: 16
  start-page: 3
  year: 2010
  ident: 10.1016/j.ddmod.2017.02.009_bib0010
  article-title: Recent advances in cancer therapy: an overview
  publication-title: Curr Pharm Des
  doi: 10.2174/138161210789941847
  contributor:
    fullname: Urruticoechea
– volume: 525
  start-page: 162
  year: 2013
  ident: 10.1016/j.ddmod.2017.02.009_bib0025
  article-title: History of gene therapy
  publication-title: Gene
  doi: 10.1016/j.gene.2013.03.137
  contributor:
    fullname: Wirth
– volume: 33
  start-page: 73
  year: 2015
  ident: 10.1016/j.ddmod.2017.02.009_bib0160
  article-title: Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
  publication-title: Nat Biotechnol
  doi: 10.1038/nbt.3081
  contributor:
    fullname: Zuris
– volume: 24
  start-page: 132
  year: 2014
  ident: 10.1016/j.ddmod.2017.02.009_bib0075
  article-title: Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases
  publication-title: Genome Res
  doi: 10.1101/gr.162339.113
  contributor:
    fullname: Cho
– volume: 21
  start-page: 704
  year: 2010
  ident: 10.1016/j.ddmod.2017.02.009_bib0110
  article-title: Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors
  publication-title: Hum Gene Ther
  doi: 10.1089/hum.2009.182
  contributor:
    fullname: Boutin
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Snippet Gene therapy has great potential for use in safe and effective cancer treatments, as it can target oncogenic pathways at a molecular level. The recent...
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Title Cancer gene therapy: innovations in therapeutic delivery of CRISPR-Cas9
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