Research graph
References from Genetically engineered hypoimmunogenic iPSCs: Strategies and considerations for clinical applications. Local targets link to admitted publications; unresolved targets remain external evidence.
iPS cell therapy 2.0: preparing for next-generation regenerative medicine
10.1002/bies.202400072 · 2024 · External reference
Do human leukocyte antigen-typed cellular therapeutics based on induced pluripotent stem cells make commercial sense?
10.1089/scd.2014.0136 · 2015 · External reference
Review of the current trends in clinical trials involving induced pluripotent stem cells
10.1007/s12015-021-10262-3 · 2022 · External reference
CRISPR provides acquired resistance against viruses in prokaryotes
10.1126/science.1138140 · 2007 · External reference
Diversity, classification and evolution of CRISPR-Cas systems
10.1016/j.mib.2017.05.008 · 2017 · External reference
An updated evolutionary classification of CRISPR-Cas systems
10.1038/nrmicro3569 · 2015 · External reference
Occurrence and diversity of CRISPR-Cas systems in the genus bifidobacterium
10.1371/journal.pone.0133661 · 2015 · External reference
CRISPR-Cas for genome editing: classification, mechanism, designing and applications
10.1016/j.ijbiomac.2023.124054 · 2023 · External reference
Cas3 is a single-stranded DNA nuclease and ATP-dependent helicase in the CRISPR/Cas immune system
10.1038/emboj.2011.41 · 2011 · External reference
Introducing a spectrum of long-range genomic deletions in human embryonic stem cells using type I CRISPR-Cas
10.1016/j.molcel.2019.03.014 · 2019 · External reference
CRISPR-Cas3 induces broad and unidirectional genome editing in human cells
10.1038/s41467-019-13226-x · 2019 · External reference
Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs
10.1016/j.stemcr.2023.07.007 · 2023 · External reference
Structure and genome editing of type I-B CRISPR-Cas
10.1038/s41467-024-48598-2 · 2024 · External reference
Cas11 enables genome engineering in human cells with compact CRISPR-Cas3 systems
10.1016/j.molcel.2021.12.032 · 2022 · External reference
Genome editing in mammalian cells using the CRISPR type I-D nuclease
10.1093/nar/gkab348 · 2021 · External reference
Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements
10.1038/nbt.4192 · 2018 · External reference
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
10.1038/ncomms7244 · 2015 · External reference
Precise large-fragment deletions in mammalian cells and mice generated by dCas9-controlled CRISPR/Cas3
2024 · External reference
A programmable Dual-RNA–Guided DNA endonuclease in adaptive bacterial immunity
10.1126/science.1225829 · 2012 · External reference
Review of CRISPR/Cas9 sgRNA design tools
10.1007/s12539-018-0298-z · 2018 · External reference
Multiplex genome editing to generate universal CAR T cells resistant to PD1 inhibition
10.1158/1078-0432.ccr-16-1300 · 2017 · External reference
Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system
10.1016/j.cell.2015.09.038 · 2015 · External reference
An update on CRISPR-Cas12 as a versatile tool in genome editing
10.1007/s11033-023-08239-1 · 2023 · External reference
Multiplex gene editing by CRISPR-Cpf1 using a single crRNA array
10.1038/nbt.3737 · 2017 · External reference
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
10.1038/nature17946 · 2016 · External reference
Base editing: advances and therapeutic opportunities
10.1038/s41573-020-0084-6 · 2020 · External reference
A highly specific CRISPR-Cas12j nuclease enables allele-specific genome editing
2023 · External reference
Noncanonical target-strand cytosine base editing via engineered Un1Cas12f1 platform
10.1038/s41467-025-64562-0 · 2025 · External reference
A Cas3-base editing tool for targetable in vivo mutagenesis
10.1038/s41467-023-39087-z · 2023 · External reference
CRISPR-STOP: gene silencing through base-editing-induced nonsense mutations
10.1038/nmeth.4327 · 2017 · External reference
Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
10.1038/nature24644 · 2017 · External reference
Adenine base editors catalyze cytosine conversions in human cells
10.1038/s41587-019-0254-4 · 2019 · External reference
Engineering a precise adenine base editor with minimal bystander editing
10.1038/s41589-022-01163-8 · 2023 · External reference
Precise, minimally evolved adenine base editors generated through mutation reversion analysis
10.1038/s41587-026-03045-z · 2026 · External reference
Search-and-replace genome editing without double-strand breaks or donor DNA
10.1038/s41586-019-1711-4 · 2019 · External reference
Gene knockout and knockin by zinc-finger nucleases: current status and perspectives
10.1007/s00018-012-1204-1 · 2012 · External reference
Efficient high-precision homology-directed repair-dependent genome editing by HDRobust
10.1038/s41592-023-01949-1 · 2023 · External reference
Template-jumping prime editing enables large insertion and exon rewriting in vivo
10.1038/s41467-023-39137-6 · 2023 · External reference
Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases
10.1038/s41587-022-01527-4 · 2023 · External reference
In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
10.1038/nature20565 · 2016 · External reference
In vivo blunt-end cloning through CRISPR/Cas9-facilitated non-homologous end-joining
10.1093/nar/gkv1542 · 2016 · External reference
Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration
10.1016/j.ymthe.2020.12.010 · 2021 · External reference
Homology-independent targeted insertion (HITI) enables guided CAR knock-in and efficient clinical scale CAR-T cell manufacturing
10.1186/s12943-023-01799-7 · 2023 · External reference
Homology-independent targeted insertion-mediated derivation of M1-biased macrophages harbouring Megf10 and CD3ζ from human pluripotent stem cells
10.1016/j.ebiom.2024.105390 · 2024 · External reference
Transposase-CRISPR mediated targeted integration (TransCRISTI) in the human genome
10.1038/s41598-022-07158-8 · 2022 · External reference
Molecular mechanisms in retrovirus DNA integration
10.1016/s0166-3542(97)00046-6 · 1997 · External reference
Lentiviral gene transfer into human and murine hematopoietic stem cells: size matters
10.1186/s13104-016-2118-z · 2016 · External reference
Retroviral insertional mutagenesis in humans: evidence for four genetic mechanisms promoting expansion of cell clones
10.1016/j.ymthe.2019.12.009 · 2020 · External reference
Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences
10.1371/journal.pbio.0020234 · 2004 · External reference
Integration of adeno-associated virus (AAV) and recombinant AAV vectors
10.1146/annurev.genet.37.110801.143717 · 2004 · External reference
Design and packaging of Adeno-Associated virus gene targeting vectors
10.1128/jvi.74.10.4612-4620.2000 · 2000 · External reference
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
10.1038/s41591-019-0344-3 · 2019 · External reference
Genome engineering with Cas9 and AAV repair templates generates frequent concatemeric insertions of viral vectors
10.1038/s41587-024-02171-w · 2025 · External reference
High levels of AAV vector integration into CRISPR-induced DNA breaks
10.1038/s41467-019-12449-2 · 2019 · External reference
Adeno-Associated virus vectors and stem cells: friends or foes?
10.1089/hum.2017.038 · 2017 · External reference
Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
10.1038/s41434-020-0182-4 · 2021 · External reference
Triple vectors expand AAV transfer capacity in the retina
10.1016/j.ymthe.2017.11.019 · 2018 · External reference
Mobilization of giant piggyBac transposons in the mouse genome
10.1093/nar/gkr764 · 2011 · External reference
Efficient transposition of the piggyBac (PB) transposon in Mammalian cells and mice
10.1016/j.cell.2005.07.013 · 2005 · External reference
Sleeping Beauty, a wide host-range transposon vector for genetic transformation in vertebrates1
10.1006/jmbi.2000.4047 · 2000 · External reference
Transposon-mediated genome manipulation in vertebrates
10.1038/nmeth.1332 · 2009 · External reference
Chromatin states shape insertion profiles of the piggyBac, Tol2 and sleeping Beauty transposons and murine leukemia virus
10.1038/srep43613 · 2017 · External reference
Structural basis of TnsC oligomerization and transposase recruitment in type I-B CRISPR-associated transposons
10.1093/nar/gkaf149 · 2025 · External reference
Molecular mechanism for Tn7-like transposon recruitment by a type I-B CRISPR effector
10.1016/j.cell.2023.07.010 · 2023 · External reference
Targeted DNA integration in human cells without double-strand breaks using CRISPR-associated transposases
10.1038/s41587-023-01748-1 · 2024 · External reference
CRISPR RNA-guided integrases for high-efficiency, multiplexed bacterial genome engineering
10.1038/s41587-020-00745-y · 2021 · External reference
RNA-guided DNA insertion with CRISPR-associated transposases
10.1126/science.aax9181 · 2019 · External reference
Structures of the holo CRISPR RNA-guided transposon integration complex
10.1038/s41586-022-05573-5 · 2023 · External reference
Precise cut-and-paste DNA insertion using engineered type V-K CRISPR-associated transposases
10.1038/s41587-022-01574-x · 2023 · External reference
Delivery of CRISPR-Cas tools for in vivo genome editing therapy: trends and challenges
10.1016/j.jconrel.2022.01.013 · 2022 · External reference
The delivery challenge: fulfilling the promise of therapeutic genome editing
10.1038/s41587-020-0565-5 · 2020 · External reference
Unresolved reference
External reference
Immunological considerations and challenges for regenerative cellular therapies
10.1038/s42003-021-02237-4 · 2021 · External reference
PVR (CD155) and Nectin-2 (CD112) as ligands of the human DNAM-1 (CD226) activating receptor: involvement in tumor cell lysis
10.1016/j.molimm.2004.07.028 · 2005 · External reference
The role of NK cell recognition of nectin and nectin-like proteins in tumor immunosurveillance
10.1016/j.semcancer.2006.07.002 · 2006 · External reference
Loss of DNAM-1 ligand expression by acute myeloid leukemia cells renders them resistant to NK cell killing
10.1080/2162402x.2016.1196308 · 2016 · External reference
A pro‐survival role for the intracellular granzyme B inhibitor Serpinb9 in natural killer cells during poxvirus infection
10.1038/icb.2017.59 · 2017 · External reference
Serpin B9 controls tumor cell killing by CAR T cells
10.1136/jitc-2022-006364 · 2023 · External reference
Protection of cell therapeutics from antibody-mediated killing by CD64 overexpression
10.1038/s41587-022-01540-7 · 2023 · External reference
Engineered off-the-shelf therapeutic T cells resist host immune rejection
10.1038/s41587-020-0601-5 · 2021 · External reference
Safety and efficacy of FT522, a First-in-Class, multi-antigen targeted, Off-the-Shelf, iPSC-Derived CD19 CAR NK cell therapy with Alloimmune Defense Receptor (ADR) in Relapsed/Refractory B-Cell lymphoma
10.1182/blood-2024-198684 · 2024 · External reference
Natural killer cells and cancer: regulation by the killer cell Ig-like receptors (KIR)
10.4161/cbt.8.23.10455 · 2009 · External reference
Activating killer cell immunoglobulin‐like receptors: detection, function and therapeutic use
10.1111/iji.12461 · 2020 · External reference
HLA-C is the inhibitory ligand that determines dominant resistance to lysis by NK1- and NK2-specific natural killer cells
10.1073/pnas.90.24.12000 · 1993 · External reference
P58 molecules as putative receptors for major histocompatibility complex (MHC) class I molecules in human natural killer (NK) cells. Anti-p58 antibodies reconstitute lysis of MHC class I-protected cells in NK clones displaying different specificities
10.1084/jem.178.2.597 · 1993 · External reference
Skin graft rejection by beta 2-microglobulin-deficient mice
10.1084/jem.175.4.885 · 1992 · External reference
Missing self triggers NK cell-mediated chronic vascular rejection of solid organ transplants
10.1038/s41467-019-13113-5 · 2019 · External reference
Genetically engineered hypoimmunogenic cell therapy
10.1038/s44222-024-00219-9 · 2024 · External reference
Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application
10.1016/j.omtm.2022.05.010 · 2022 · External reference
A clinical-grade HLA haplobank of human induced pluripotent stem cells matching approximately 40% of the Japanese population
2023 · External reference
Safety assessment of genome editing in human gene therapy products using next-generation sequencing
2026 · External reference
Hotspots of Human mutation
10.1016/j.tig.2020.10.003 · 2021 · External reference
Copy number variation: new insights in genome diversity
10.1101/gr.3677206 · 2006 · External reference
The COSMIC Cancer Gene Census: describing genetic dysfunction across all human cancers
10.1038/s41568-018-0060-1 · 2018 · External reference
Current perspective on evaluation of tumorigenicity of cellular and tissue-based products derived from induced pluripotent
2013 · External reference
Distinctive clinical and pathologic features of immature teratomas arising from induced pluripotent stem cell-derived beta cell injection in a diabetes patient
10.1089/scd.2021.0255 · 2022 · External reference
A first-in-human clinical study of an allogenic iPSC-derived corneal endothelial cell substitute transplantation for bullous keratopathy
2025 · External reference
Next-generation sequencing for constitutional variants in the clinical laboratory, 2021 revision: a technical standard of the American College of Medical Genetics and Genomics (ACMG)
2021 · External reference
The status of the human gene catalogue
10.1038/s41586-023-06490-x · 2023 · External reference
The sequence of the human genome
10.1126/science.1058040 · 2001 · External reference
Comprehensive assessment of on- and off-target mutagenesis via lipid nanoparticle delivery of CRISPR-Cas9 genome editing
10.1016/j.omtn.2026.102958 · 2026 · External reference
Whole genome and exome sequencing reference datasets from a multi-center and cross-platform benchmark study
10.1038/s41597-021-01077-5 · 2021 · External reference
Analyses of non-coding somatic drivers in 2,658 cancer whole genomes
10.1038/s41586-020-1965-x · 2020 · External reference
Pan-cancer analysis of non-coding recurrent mutations and their possible involvement in cancer pathogenesis
10.1093/narcan/zcab008 · 2021 · External reference
Comparison of exome and genome sequencing technologies for the complete capture of protein-coding regions
10.1002/humu.22813 · 2015 · External reference
Systematic dissection of biases in whole-exome and whole-genome sequencing reveals major determinants of coding sequence coverage
10.1038/s41598-020-59026-y · 2020 · External reference
Unintended CRISPR-Cas9 editing outcomes: a review of the detection and prevalence of structural variants generated by gene-editing in human cells
10.1007/s00439-023-02561-1 · 2023 · External reference
Mechanisms for structural variation in the human genome
10.1007/s40142-013-0012-8 · 2013 · External reference
Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing
10.1126/sciadv.abo7676 · 2022 · External reference
Whole genomic analysis reveals atypical non-homologous off-target large structural variants induced by CRISPR-Cas9-mediated genome editing
10.1038/s41467-023-40901-x · 2023 · External reference
CRISPR-Cas9 induces large structural variants at on-target and off-target sites in vivo that segregate across generations
10.1038/s41467-022-28244-5 · 2022 · External reference
De novo CNV formation in mouse embryonic stem cells occurs in the absence of Xrcc4-dependent nonhomologous end joining
10.1371/journal.pgen.1002981 · 2012 · External reference
Mechanisms underlying structural variant formation in genomic disorders
10.1038/nrg.2015.25 · 2016 · External reference
Points to consider in the development of seed stocks of pluripotent stem cells for clinical applications: international Stem Cell Banking Initiative (ISCBI), Regen
2015 · External reference
Immune responses to viral gene therapy vectors
10.1016/j.ymthe.2020.01.001 · 2020 · External reference
Identification of preexisting adaptive immunity to Cas9 proteins in humans
10.1038/s41591-018-0326-x · 2019 · External reference
Cas9 immunity creates challenges for CRISPR gene editing therapies
10.1038/s41467-018-05843-9 · 2018 · External reference
PCR-based analytics of gene therapies using adeno-associated virus vectors: considerations for cGMP method development
10.1016/j.omtm.2023.101132 · 2023 · External reference
Clearance of residual genome editing components used for ex vivo genome-editing of allogeneic cell therapy products
10.1016/j.jcyt.2024.06.005 · 2024 · External reference
Accurate quantification and characterization of Adeno-Associated viral vectors
10.3389/fmicb.2019.01570 · 2019 · External reference
Safety confirmation of induced pluripotent stem cell-derived cardiomyocyte patch transplantation for ischemic cardiomyopathy: first three case reports
10.3389/fcvm.2023.1182209 · 2023 · External reference
Phase I/II trial of iPS-cell-derived dopaminergic cells for Parkinson's disease
10.1038/s41586-025-08700-0 · 2025 · External reference
POS0056 CNTY-101, AN iPSC-DERIVED ALLOGENEIC CD19 TARGETING CAR-NK PRODUCT, EXHIBITS ROBUST B CELL DEPLETION AND HAS BROAD POTENTIAL FOR DEVELOPMENT IN B CELL-DRIVEN AUTOIMMUNE DISEASES
10.1016/j.ard.2025.05.453 · 2025 · External reference
1318-OR: CNTY-813: scalable production of allo-evasion 5.0-Engineered IPSC beta islets for off-the-shelf cell therapies
10.2337/db26-1318-or · 2026 · External reference
Survival of Transplanted allogeneic beta cells with no immunosuppression
10.1056/nejmoa2503822 · 2025 · External reference
How safe are universal pluripotent stem cells?
10.1016/j.stem.2020.02.006 · 2020 · External reference
Is hypoimmunogenic stem cell therapy safe in times of pandemics?
10.1016/j.stemcr.2022.02.014 · 2022 · External reference
Linking a cell-division gene and a suicide gene to define and improve cell therapy safety
10.1038/s41586-018-0733-7 · 2018 · External reference
Metabolic engineering generates a transgene-free safety switch for cell therapy
10.1038/s41587-020-0580-6 · 2020 · External reference
iPS cell therapy 2.0: preparing for next-generation regenerative medicine
10.1002/bies.202400072 · ExternalCitation · doi-reference
Comparison of exome and genome sequencing technologies for the complete capture of protein-coding regions
10.1002/humu.22813 · ExternalCitation · doi-reference
Sleeping Beauty, a wide host-range transposon vector for genetic transformation in vertebrates1
10.1006/jmbi.2000.4047 · ExternalCitation · doi-reference
Gene knockout and knockin by zinc-finger nucleases: current status and perspectives
10.1007/s00018-012-1204-1 · ExternalCitation · doi-reference
Unintended CRISPR-Cas9 editing outcomes: a review of the detection and prevalence of structural variants generated by gene-editing in human cells
10.1007/s00439-023-02561-1 · ExternalCitation · doi-reference
An update on CRISPR-Cas12 as a versatile tool in genome editing
10.1007/s11033-023-08239-1 · ExternalCitation · doi-reference
Review of the current trends in clinical trials involving induced pluripotent stem cells
10.1007/s12015-021-10262-3 · ExternalCitation · doi-reference
Review of CRISPR/Cas9 sgRNA design tools
10.1007/s12539-018-0298-z · ExternalCitation · doi-reference
Mechanisms for structural variation in the human genome
10.1007/s40142-013-0012-8 · ExternalCitation · doi-reference
POS0056 CNTY-101, AN iPSC-DERIVED ALLOGENEIC CD19 TARGETING CAR-NK PRODUCT, EXHIBITS ROBUST B CELL DEPLETION AND HAS BROAD POTENTIAL FOR DEVELOPMENT IN B CELL-DRIVEN AUTOIMMUNE DISEASES
10.1016/j.ard.2025.05.453 · ExternalCitation · doi-reference
Efficient transposition of the piggyBac (PB) transposon in Mammalian cells and mice
10.1016/j.cell.2005.07.013 · ExternalCitation · doi-reference
Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system
10.1016/j.cell.2015.09.038 · ExternalCitation · doi-reference
Molecular mechanism for Tn7-like transposon recruitment by a type I-B CRISPR effector
10.1016/j.cell.2023.07.010 · ExternalCitation · doi-reference
Homology-independent targeted insertion-mediated derivation of M1-biased macrophages harbouring Megf10 and CD3ζ from human pluripotent stem cells
10.1016/j.ebiom.2024.105390 · ExternalCitation · doi-reference
CRISPR-Cas for genome editing: classification, mechanism, designing and applications
10.1016/j.ijbiomac.2023.124054 · ExternalCitation · doi-reference
Delivery of CRISPR-Cas tools for in vivo genome editing therapy: trends and challenges
10.1016/j.jconrel.2022.01.013 · ExternalCitation · doi-reference
Clearance of residual genome editing components used for ex vivo genome-editing of allogeneic cell therapy products
10.1016/j.jcyt.2024.06.005 · ExternalCitation · doi-reference
Diversity, classification and evolution of CRISPR-Cas systems
10.1016/j.mib.2017.05.008 · ExternalCitation · doi-reference
Introducing a spectrum of long-range genomic deletions in human embryonic stem cells using type I CRISPR-Cas
10.1016/j.molcel.2019.03.014 · ExternalCitation · doi-reference
Cas11 enables genome engineering in human cells with compact CRISPR-Cas3 systems
10.1016/j.molcel.2021.12.032 · ExternalCitation · doi-reference
PVR (CD155) and Nectin-2 (CD112) as ligands of the human DNAM-1 (CD226) activating receptor: involvement in tumor cell lysis
10.1016/j.molimm.2004.07.028 · ExternalCitation · doi-reference
Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application
10.1016/j.omtm.2022.05.010 · ExternalCitation · doi-reference
PCR-based analytics of gene therapies using adeno-associated virus vectors: considerations for cGMP method development
10.1016/j.omtm.2023.101132 · ExternalCitation · doi-reference
Comprehensive assessment of on- and off-target mutagenesis via lipid nanoparticle delivery of CRISPR-Cas9 genome editing
10.1016/j.omtn.2026.102958 · ExternalCitation · doi-reference
The role of NK cell recognition of nectin and nectin-like proteins in tumor immunosurveillance
10.1016/j.semcancer.2006.07.002 · ExternalCitation · doi-reference
How safe are universal pluripotent stem cells?
10.1016/j.stem.2020.02.006 · ExternalCitation · doi-reference
Is hypoimmunogenic stem cell therapy safe in times of pandemics?
10.1016/j.stemcr.2022.02.014 · ExternalCitation · doi-reference
Dual CRISPR-Cas3 system for inducing multi-exon skipping in DMD patient-derived iPSCs
10.1016/j.stemcr.2023.07.007 · ExternalCitation · doi-reference
Hotspots of Human mutation
10.1016/j.tig.2020.10.003 · ExternalCitation · doi-reference
Triple vectors expand AAV transfer capacity in the retina
10.1016/j.ymthe.2017.11.019 · ExternalCitation · doi-reference
Retroviral insertional mutagenesis in humans: evidence for four genetic mechanisms promoting expansion of cell clones
10.1016/j.ymthe.2019.12.009 · ExternalCitation · doi-reference
Immune responses to viral gene therapy vectors
10.1016/j.ymthe.2020.01.001 · ExternalCitation · doi-reference
Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration
10.1016/j.ymthe.2020.12.010 · ExternalCitation · doi-reference
Molecular mechanisms in retrovirus DNA integration
10.1016/s0166-3542(97)00046-6 · ExternalCitation · doi-reference
Cas3 is a single-stranded DNA nuclease and ATP-dependent helicase in the CRISPR/Cas immune system
10.1038/emboj.2011.41 · ExternalCitation · doi-reference
A pro‐survival role for the intracellular granzyme B inhibitor Serpinb9 in natural killer cells during poxvirus infection
10.1038/icb.2017.59 · ExternalCitation · doi-reference
Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
10.1038/nature17946 · ExternalCitation · doi-reference
In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration
10.1038/nature20565 · ExternalCitation · doi-reference
Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
10.1038/nature24644 · ExternalCitation · doi-reference
Multiplex gene editing by CRISPR-Cpf1 using a single crRNA array
10.1038/nbt.3737 · ExternalCitation · doi-reference
Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements
10.1038/nbt.4192 · ExternalCitation · doi-reference
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
10.1038/ncomms7244 · ExternalCitation · doi-reference
Transposon-mediated genome manipulation in vertebrates
10.1038/nmeth.1332 · ExternalCitation · doi-reference
CRISPR-STOP: gene silencing through base-editing-induced nonsense mutations
10.1038/nmeth.4327 · ExternalCitation · doi-reference
Mechanisms underlying structural variant formation in genomic disorders
10.1038/nrg.2015.25 · ExternalCitation · doi-reference
An updated evolutionary classification of CRISPR-Cas systems
10.1038/nrmicro3569 · ExternalCitation · doi-reference
Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
10.1038/s41434-020-0182-4 · ExternalCitation · doi-reference
Cas9 immunity creates challenges for CRISPR gene editing therapies
10.1038/s41467-018-05843-9 · ExternalCitation · doi-reference
High levels of AAV vector integration into CRISPR-induced DNA breaks
10.1038/s41467-019-12449-2 · ExternalCitation · doi-reference
Missing self triggers NK cell-mediated chronic vascular rejection of solid organ transplants
10.1038/s41467-019-13113-5 · ExternalCitation · doi-reference
CRISPR-Cas3 induces broad and unidirectional genome editing in human cells
10.1038/s41467-019-13226-x · ExternalCitation · doi-reference
CRISPR-Cas9 induces large structural variants at on-target and off-target sites in vivo that segregate across generations
10.1038/s41467-022-28244-5 · ExternalCitation · doi-reference
A Cas3-base editing tool for targetable in vivo mutagenesis
10.1038/s41467-023-39087-z · ExternalCitation · doi-reference
Template-jumping prime editing enables large insertion and exon rewriting in vivo
10.1038/s41467-023-39137-6 · ExternalCitation · doi-reference
Whole genomic analysis reveals atypical non-homologous off-target large structural variants induced by CRISPR-Cas9-mediated genome editing
10.1038/s41467-023-40901-x · ExternalCitation · doi-reference
Structure and genome editing of type I-B CRISPR-Cas
10.1038/s41467-024-48598-2 · ExternalCitation · doi-reference
Noncanonical target-strand cytosine base editing via engineered Un1Cas12f1 platform
10.1038/s41467-025-64562-0 · ExternalCitation · doi-reference
The COSMIC Cancer Gene Census: describing genetic dysfunction across all human cancers
10.1038/s41568-018-0060-1 · ExternalCitation · doi-reference
Base editing: advances and therapeutic opportunities
10.1038/s41573-020-0084-6 · ExternalCitation · doi-reference
Linking a cell-division gene and a suicide gene to define and improve cell therapy safety
10.1038/s41586-018-0733-7 · ExternalCitation · doi-reference
Search-and-replace genome editing without double-strand breaks or donor DNA
10.1038/s41586-019-1711-4 · ExternalCitation · doi-reference
Analyses of non-coding somatic drivers in 2,658 cancer whole genomes
10.1038/s41586-020-1965-x · ExternalCitation · doi-reference
Structures of the holo CRISPR RNA-guided transposon integration complex
10.1038/s41586-022-05573-5 · ExternalCitation · doi-reference
The status of the human gene catalogue
10.1038/s41586-023-06490-x · ExternalCitation · doi-reference
Phase I/II trial of iPS-cell-derived dopaminergic cells for Parkinson's disease
10.1038/s41586-025-08700-0 · ExternalCitation · doi-reference
Adenine base editors catalyze cytosine conversions in human cells
10.1038/s41587-019-0254-4 · ExternalCitation · doi-reference
CRISPR RNA-guided integrases for high-efficiency, multiplexed bacterial genome engineering
10.1038/s41587-020-00745-y · ExternalCitation · doi-reference
The delivery challenge: fulfilling the promise of therapeutic genome editing
10.1038/s41587-020-0565-5 · ExternalCitation · doi-reference
Metabolic engineering generates a transgene-free safety switch for cell therapy
10.1038/s41587-020-0580-6 · ExternalCitation · doi-reference
Engineered off-the-shelf therapeutic T cells resist host immune rejection
10.1038/s41587-020-0601-5 · ExternalCitation · doi-reference
Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases
10.1038/s41587-022-01527-4 · ExternalCitation · doi-reference
Protection of cell therapeutics from antibody-mediated killing by CD64 overexpression
10.1038/s41587-022-01540-7 · ExternalCitation · doi-reference
Precise cut-and-paste DNA insertion using engineered type V-K CRISPR-associated transposases
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10.1080/2162402x.2016.1196308 · ExternalCitation · doi-reference
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10.1084/jem.178.2.597 · ExternalCitation · doi-reference
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