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Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus

Nature biotechnology, 2022-01, Vol.40 (1), p.94-102

2022

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In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

Nature communications, 2017-02, Vol.8 (1), p.14500-14500, Article 14500

2017

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Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration

Genome research, 2017-03, Vol.27 (3), p.419

2017

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CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

Nature communications, 2018-05, Vol.9 (1), p.1855-8, Article 1855

2018

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Measuring and Reducing Off-Target Activities of Programmable Nucleases Including CRISPR-Cas9

Molecules and Cells, 2015, 38(6), , pp.475-481

2015

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Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

Molecular therapy, 2018-06, Vol.26 (6), p.1529-1538

2018

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Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery

Experimental and Molecular Medicine, 2023, 55(0), , pp.377-384

2023

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Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus

Molecular therapy, 2019-01, Vol.27 (1), p.130-136

2019

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Expanded targeting scope of LbCas12a variants allows editing of multiple oncogenic mutations

Molecular therapy. Nucleic acids, 2022-12, Vol.30, p.131-142

2022

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CRISPR technologies for the treatment of Duchenne muscular dystrophy

Molecular therapy, 2021-11, Vol.29 (11), p.3179-3191

2021

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CRISPR base editing-mediated correction of a tau mutation rescues cognitive decline in a mouse model of tauopathy

Translational neurodegeneration, 2024-04, Vol.13 (1), p.21-21

2024

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Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog

The journal of gene medicine, 2011-09, Vol.13 (9), p.497-506

2011

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Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in

Human gene therapy, 2013-07, Vol.24 (7), p.692

2013

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Correction to: Recent advances in CRISPR technologies for genome editing

Archives of pharmacal research, 2021-08, Vol.44 (8), p.836-838

2021

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Recent advances in CRISPR technologies for genome editing

Archives of Pharmacal Research, 2021, 44(6), , pp.537-552

2021

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Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

Nature biotechnology, 2018-07, Vol.36 (6), p.536-539

2018

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Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy

Human gene therapy, 2013-05, Vol.24 (5), p.479

2013

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Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice

Human gene therapy, 2014-02, Vol.25 (2), p.98

2014

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Selective disruption of an oncogenic mutant allele by CRISPR/Cas9 induces efficient tumor regression

Nucleic acids research, 2017-07, Vol.45 (13), p.7897

2017

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Therapeutic applications of CRISPR RNA-guided genome editing

Briefings in functional genomics, 2017-01, Vol.16 (1), p.38-45

2017

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Abstract 861: Precise excision of an oncogenic allele of tumor by Adenovirus-based CRISPR/Cas9 system induces tumor regression

Cancer research (Chicago, Ill.), 2019-07, Vol.79 (13_Supplement), p.861-861

2019

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CRISPR-Cas12a with an oAd Induces Precise and Cancer-Specific Genomic Reprogramming of EGFR and Efficient Tumor Regression

Molecular therapy, 2020-10, Vol.28 (10), p.2286-2296

2020

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Survivin enhances hippocampal neurogenesis and cognitive function in Alzheimer's disease mouse model

CNS neuroscience & therapeutics, 2023-10, Vol.30 (4), p.e14509-e14509

2023

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Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

Measuring and Reducing Off-Target Activities of Programmable Nucleases Including CRISPR-Cas9

Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

Targeted dual base editing with Campylobacter jejuni Cas9 by single AAV-mediated delivery

Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus

Expanded targeting scope of LbCas12a variants allows editing of multiple oncogenic mutations

CRISPR technologies for the treatment of Duchenne muscular dystrophy

CRISPR base editing-mediated correction of a tau mutation rescues cognitive decline in a mouse model of tauopathy

Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog

Gene correction of a duchenne muscular dystrophy mutation by meganuclease-enhanced exon knock-in

Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases

Correction to: Recent advances in CRISPR technologies for genome editing

Recent advances in CRISPR technologies for genome editing

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy

Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice

Selective disruption of an oncogenic mutant allele by CRISPR/Cas9 induces efficient tumor regression

Therapeutic applications of CRISPR RNA-guided genome editing

Abstract 861: Precise excision of an oncogenic allele of tumor by Adenovirus-based CRISPR/Cas9 system induces tumor regression

CRISPR-Cas12a with an oAd Induces Precise and Cancer-Specific Genomic Reprogramming of EGFR and Efficient Tumor Regression

Survivin enhances hippocampal neurogenesis and cognitive function in Alzheimer's disease mouse model

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