Keywords
To read this article in full you will need to make a payment
Purchase one-time access:
Academic & Personal: 24 hour online accessCorporate R&D Professionals: 24 hour online accessOne-time access price info
- For academic or personal research use, select 'Academic and Personal'
- For corporate R&D use, select 'Corporate R&D Professionals'
Subscribers receive full online access to your subscription and archive of back issues up to and including 2002.
Content published before 2002 is available via pay-per-view purchase only.
Subscribe:
Subscribe to Clinics in Laboratory MedicineAlready a print subscriber? Claim online access
Already an online subscriber? Sign in
Register: Create an account
Institutional Access: Sign in to ScienceDirect
References
- A decade’s perspective on DNA sequencing technology.Nature. 2011; 470: 198-203
- Estimating the burden and economic impact of pediatric genetic disease.Genet Med. 2019; 21: 1781-1789
- A restriction enzyme from Hemophilus influenzae. I. Purification and general properties.J Mol Biol. 1970; 51: 379-391
- A restriction enzyme from Hemophilus influenzae. II.J Mol Biol. 1970; 51: 393-409
- Altering the genome by homologous recombination.Science. 1989; 244: 1288-1292
- Recombination in mouse L cells between DNA introduced into cells and homologous chromosomal sequences.Proc Natl Acad Sci U S A. 1985; 82: 1391-1395
- Genetic and physical analysis of double-strand break repair and recombination in Saccharomyces cerevisiae.Genetics. 1989; 122: 519-534
- Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases.Genetics. 2002; 161: 1169-1175
- DNA breakage and repair.Adv Genet. 1998; 38: 185-218
- Zinc fingers: a novel protein fold for nucleic acid recognition.Cold Spring Harb Symp Quant Biol. 1987; 52: 473-482
- An improved zinc-finger nuclease architecture for highly specific genome editing.Nat Biotechnol. 2007; 25: 778-785
- Chimeric nucleases stimulate gene targeting in human cells.Science. 2003; 300: 763
- Breaking the code of DNA binding specificity of TAL-type III effectors.Science. 2009; 326: 1509-1512
- Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes.Nucleic Acids Res. 2011; 39: 6315-6325
- Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product.J Bacteriol. 1987; 169: 5429-5433
- RNA-guided genetic silencing systems in bacteria and archaea.Nature. 2012; 482: 331-338
- The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli.Nucleic Acids Res. 2011; 39: 9275-9282
- Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements.J Mol Evol. 2005; 60: 174-182
- Small CRISPR RNAs guide antiviral defense in prokaryotes.Science. 2008; 321: 960-964
- Phage response to CRISPR-encoded resistance in Streptococcus thermophilus.J Bacteriol. 2008; 190: 1390-1400
- A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.Science. 2012; 337: 816-821
- Multiplex genome engineering using CRISPR/Cas systems.Science. 2013; 339: 819-823
- RNA-guided human genome engineering via Cas9.Science. 2013; 339: 823-826
- Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage.Nature. 2016; 533: 420-424
- A new class of medicines through DNA editing.N Engl J Med. 2019; 380: 947-959
- Predicting the mutations generated by repair of Cas9-induced double-strand breaks.Nat Biotechnol. 2019; 37: 64-72
- RNA editing with CRISPR-Cas13.Science. 2017; 358: 1019-1027
- Simple knockout by electroporation of engineered endonucleases into intact rat embryos.Sci Rep. 2014; 4: 6382
- Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.N Engl J Med. 2014; 370: 901-910
- Preclinical development and qualification of ZFN-mediated CCR5 disruption in human hematopoietic stem/progenitor cells.Mol Ther Methods Clin Dev. 2016; 3: 16067
- Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells.Sci Transl Med. 2017; 9https://doi.org/10.1126/scitranslmed.aaj2013
- Multiplex genome-edited T-cell manufacturing platform for “off-the-shelf” adoptive T-cell immunotherapies.Cancer Res. 2015; 75: 3853-3864
- CRISPR/CAS9-Mediated genome editing of miRNA-155 inhibits proinflammatory cytokine production by RAW264.7 cells.Biomed Res Int. 2015; 2015: 326042
- Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.JAMA. 2015; 313: 1550-1563
- Treating B-cell cancer with T cells expressing anti-CD19 chimeric antigen receptors.Nat Rev Clin Oncol. 2013; 10: 267-276
- CAR T cell immunotherapy for human cancer.Science. 2018; 359: 1361-1365
- Chimeric antigen receptor T cells for sustained remissions in leukemia.N Engl J Med. 2014; 371: 1507-1517
- Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection.Nature. 2017; 543: 113-117
- Reprogramming human T cell function and specificity with non-viral genome targeting.Nature. 2018; 559: 405-409
- Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Blood. 2016; 127: 839-848
- Sickle cell disease: new opportunities and challenges in Africa.ScientificWorldJournal. 2013; 2013: 193252
- Production of gene-corrected adult beta globin protein in human erythrocytes differentiated from patient iPSCs after genome editing of the sickle point mutation.Stem Cells. 2015; 33: 1470-1479
- BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis.Nature. 2015; 527: 192-197
- Genome editing for sickle cell disease: a little BCL11A goes a long way.Mol Ther. 2017; 25: 561-562
- Advances in CRISPR/Cas9 technology for in vivo translation.Biol Pharm Bull. 2019; 42: 304-311
- Non-viral CRISPR/Cas gene editing in vitro and in vivo enabled by synthetic nanoparticle Co-delivery of Cas9 mRNA and sgRNA.Angew Chem Int Ed. 2017; 56: 1059-1063
- A split-Cas9 architecture for inducible genome editing and transcription modulation.Nat Biotechnol. 2015; 33: 139-142
- Off-target effects in CRISPR/Cas9-mediated genome engineering.Mol Ther Nucleic Acids. 2015; 4: e264
- Immune responses to gene therapy vectors: influence on vector function and effector mechanisms.Gene Ther. 2004; 11: S10-S17
- AAV-mediated gene therapy for research and therapeutic purposes.Annu Rev Virol. 2014; 1: 427-451
- Adenovirus-mediated gene delivery: potential applications for gene and cell-based therapies in the new era of personalized medicine.Genes Dis. 2017; 4: 43-63
- Engineered viruses as genome editing devices.Mol Ther. 2016; 24: 447-457
- Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells.Nat Biotechnol. 2015; 33: 543-548
- Site-specific integration by adeno-associated virus.Proc Natl Acad Sci U S A. 1996; 93: 11288-11294
- A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.Nat Biotechnol. 2016; 34: 334-338
- Design and development of polymers for gene delivery.Nat Rev Drug Discov. 2005; 4: 581-593
- Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors.Hum Gene Ther. 2006; 17: 253-263
- Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo.Nat Biotechnol. 2015; 33: 73-80
- In Vivo CRISPR/Cas9 gene editing corrects retinal dystrophy in the S334ter-3 rat model of autosomal dominant retinitis pigmentosa.Mol Ther. 2016; 24: 556-563
- In vivo editing of the human mutant rhodopsin gene by electroporation of plasmid-based CRISPR/Cas9 in the mouse retina.Mol Ther Nucleic Acids. 2016; 5: e389
- Translational advances of hydrofection by hydrodynamic injection.Genes. 2018; 9https://doi.org/10.3390/genes9030136
- Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.Nat Biotechnol. 2014; 32: 551-553
- Rational design of cationic lipids for siRNA delivery.Nat Biotechnol. 2010; 28: 172-176
- A collaborative assembly strategy for tumor-targeted siRNA delivery.J Am Chem Soc. 2015; 137: 6000-6010
- Artificial virus delivers CRISPR-Cas9 system for genome editing of cells in mice.ACS Nano. 2017; 11: 95-111
- Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair.Nat Biomed Eng. 2017; 1: 889-901
- Protein-gold nanoparticle interactions and their possible impact on biomedical applications.Acta Biomater. 2017; 55: 13-27
- Delivering CRISPR: a review of the challenges and approaches.Drug Deliv. 2018; 25: 1234-1257
- In vivo genome editing as a therapeutic approach.Int J Mol Sci. 2018; 19https://doi.org/10.3390/ijms19092721
- In Vivo applications of CRISPR-based genome editing in the retina.Front Cell Dev Biol. 2018; 6: 53
- Allele-specific CRISPR-Cas9 genome editing of the single-base P23H mutation for rhodopsin-associated dominant retinitis pigmentosa.CRISPR J. 2018; 1: 55-64
- Dystrophin: the protein product of the Duchenne muscular dystrophy locus.Cell. 1987; 51: 919-928
- Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin.Am J Hum Genet. 1995; 56: 725-731
- In vivo gene editing in dystrophic mouse muscle and muscle stem cells.Science. 2016; 351: 407-411
- Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.Science. 2016; 351: 400-403
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.Science. 2016; 351: 403-407
- Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.Nat Commun. 2017; 8: 14454
- Battling CRISPR-Cas9 off-target genome editing.Cell Biol Toxicol. 2019; 35: 403-406
- High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells.Nat Biotechnol. 2013; 31: 822-826
- DNA targeting specificity of RNA-guided Cas9 nucleases.Nat Biotechnol. 2013; 31: 827-832
- Whole genome analysis of CRISPR Cas9 sgRNA off-target homologies via an efficient computational algorithm.BMC Genomics. 2017; 18: 826
- High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity.Nat Biotechnol. 2013; 31: 839-843
- GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases.Nat Biotechnol. 2015; 33: 187-197
- Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins.Genome Res. 2014; 24: 1012-1019
- Small molecule-triggered Cas9 protein with improved genome-editing specificity.Nat Chem Biol. 2015; 11: 316-318
- Rationally engineered Cas9 nucleases with improved specificity.Science. 2016; 351: 84-88
- High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.Nature. 2016; 529: 490-495
- Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.Cell. 2013; 154: 1380-1389
- Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases.Genome Res. 2014; 24: 132-141
- Improving CRISPR-Cas nuclease specificity using truncated guide RNAs.Nat Biotechnol. 2014; 32: 279-284
- Increasing the specificity of CRISPR systems with engineered RNA secondary structures.Nat Biotechnol. 2019; 37: 657-666
- Improving CRISPR-Cas specificity with chemical modifications in single-guide RNAs.Nucleic Acids Res. 2018; 46: 792-803
- Low incidence of off-target mutations in individual CRISPR-Cas9 and TALEN targeted human stem cell clones detected by whole-genome sequencing.Cell Stem Cell. 2014; 15: 27-30
- Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs.Cell Stem Cell. 2014; 15: 12-13
- Implications of human genetic variation in CRISPR-based therapeutic genome editing.Nat Med. 2017; 23: 1095-1101
- Open-source guideseq software for analysis of GUIDE-seq data.Nat Biotechnol. 2016; 34: 483
- Defining CRISPR-Cas9 genome-wide nuclease activities with CIRCLE-seq.Nat Protoc. 2018; 13: 2615-2642
- CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR-Cas9 nuclease off-targets.Nat Methods. 2017; 14: 607-614
- Unbiased detection of CRISPR off-targets in vivo using DISCOVER-Seq.Science. 2019; 364: 286-289
- The MRN complex in double-strand break repair and telomere maintenance.FEBS Lett. 2010; 584: 3682-3695
- Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements.Nat Biotechnol. 2018; 36: 765-771
- In vivo CRISPR editing with no detectable genome-wide off-target mutations.Nature. 2018; 561: 416-419
- Patient-customized oligonucleotide therapy for a rare genetic disease.N Engl J Med. 2019; 381: 1644-1652
- Mutations in MFSD8/CLN7 are a frequent cause of variant-late infantile neuronal ceroid lipofuscinosis.Hum Mutat. 2009; 30: E530-E540
- Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.Lancet. 2016; 388: 3017-3026
- Research C for DE and. Expanded access to investigational drugs for treatment use - questions and answers. U.S. Food and Drug Administration.(Available at:) (Accessed November 29, 2019)
- Research C for BE and. Expedited programs for regenerative medicine therapies for serious conditions. U.S. Food and Drug Administration.(Available at:) (Accessed November 29, 2019)
- Research C for BE and. Regulatory considerations for human cells, tissues, and cellular and tissue-based products: minimal manipulation and homologous use. U.S. Food and Drug Administration.(Available at:) (Accessed November 29, 2019)
Article info
Identification
Copyright
© 2020 Elsevier Inc. All rights reserved.
