Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

In vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system

(2021) Qian Li et al.   Molecular Therapy-Methods & Clinical Development

Single AAV-mediated CRISPR/Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis

(2021) Jinkun Wen et al.   MOLECULAR THERAPY

Discovery and engineering of small SlugCas9 with broad targeting range and high specificity and activity

(2021) Ziying Hu et al.   NUCLEIC ACIDS RESEARCH

Versatile and robust genome editing with Streptococcus thermophilus CRISPR1-Cas9

(2020) Daniel Agudelo et al.   GENOME RESEARCH

Treatment of a Mouse Model of ALS by In Vivo Base Editing

(2020) Colin K.W. Lim et al.   MOLECULAR THERAPY

The promise and challenge of therapeutic genome editing

(2020) Jennifer A. Doudna   NATURE

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

(2020) Jonathan M. Levy et al.   Nature Biomedical Engineering

Anti-CRISPRs: Protein Inhibitors of CRISPR-Cas Systems

(2020) Alan R. Davidson et al.   Annual Review of Biochemistry

DolphinNext: a distributed data processing platform for high throughput genomics

(2020) Onur Yukselen et al.   BMC GENOMICS

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

(2020) Dan Wang et al.   CELL

Anti-CRISPR protein applications: natural brakes for CRISPR-Cas technologies

(2020) Nicole D. Marino et al.   NATURE METHODS

A compact Cas9 ortholog from Staphylococcus Auricularis (SauriCas9) expands the DNA targeting scope

(2020) Ziying Hu et al.   PLOS BIOLOGY

Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

(2020) James M. Wilson et al.   HUMAN GENE THERAPY

Induced liver regeneration enhances CRISPR/Cas9-mediated gene repair in tyrosinemia type 1

(2020) Qingshuo Zhang et al.   HUMAN GENE THERAPY

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Unveiling prognostics biomarkers of tyrosine metabolism reprogramming in liver cancer by cross-platform gene expression analyses

(2020) Tran N. Nguyen et al.   PLoS One

In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness

(2020) Wei-Hsi Yeh et al.   Science Translational Medicine

Rational selection of CRISPR/Cas9 guide RNAs for homology directed genome editing

(2020) Kristina J. Tatiossian et al.   MOLECULAR THERAPY

Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing

(2020) Minh Thuan Nguyen Tran et al.   Nature Communications

NanoGalaxy: Nanopore long-read sequencing data analysis in Galaxy

(2020) Willem de Koning et al.   GigaScience

AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

(2020) Ngoc Tam Tran et al.   Molecular Therapy-Methods & Clinical Development

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

CasX enzymes comprise a distinct family of RNA-guided genome editors

(2019) Jun-Jie Liu et al.   NATURE

Precise therapeutic gene correction by a simple nuclease-induced double-stranded break

(2019) Sukanya Iyer et al.   NATURE

Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations

(2019) Reza Shahbazi et al.   NATURE MATERIALS

Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses

(2019) Donna J. Palmer et al.   Molecular Therapy-Methods & Clinical Development

Utility of self-destructing CRISPR/Cas constructs for targeted gene editing in the retina

(2019) Fan Li et al.   HUMAN GENE THERAPY

Tissue-restricted Genome Editing in vivo Specified by MicroRNA-repressible Anti-CRISPR Proteins

(2019) Jooyoung Lee et al.   RNA

Targeted exon skipping with AAV-mediated split adenine base editors

(2019) Jackson Winter et al.   Cell Discovery

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States

(2019) Wei Sun et al.   MOLECULAR CELL

Advances in genome editing through control of DNA repair pathways

(2019) Charles D. Yeh et al.   NATURE CELL BIOLOGY

AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

(2019) Daelyn Y. Richards et al.   Molecular Therapy-Methods & Clinical Development

UDiTaS™, a genome editing detection method for indels and genome rearrangements

(2018) Georgia Giannoukos et al.   BMC GENOMICS

CRISPR RNAs trigger innate immune responses in human cells

(2018) Sojung Kim et al.   GENOME RESEARCH

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

(2018) Lili Wang et al.   NATURE BIOTECHNOLOGY

The Galaxy platform for accessible, reproducible and collaborative biomedical analyses: 2018 update

(2018) Enis Afgan et al.   NUCLEIC ACIDS RESEARCH

Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras

(2018) Phillip W.L. Tai et al.   Molecular Therapy-Methods & Clinical Development

Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

(2018) Dan Wang et al.   NATURE BIOTECHNOLOGY

A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing

(2018) Alireza Edraki et al.   MOLECULAR CELL

Predictable and precise template-free CRISPR editing of pathogenic variants

(2018) Max W. Shen et al.   NATURE

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

(2018) Lukas Villiger et al.   NATURE MEDICINE

Base editing: precision chemistry on the genome and transcriptome of living cells

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

Orthogonal Cas9–Cas9 chimeras provide a versatile platform for genome editing

(2018) Mehmet Fatih Bolukbasi et al.   Nature Communications

Viral vector-based delivery of CRISPR/Cas9 and donor DNA for homology directed repair in an in vitro model for canine hemophilia B

(2018) Jian Gao et al.   Molecular Therapy-Nucleic Acids

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

(2018) Ang Li et al.   Molecular Therapy-Methods & Clinical Development

Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity

(2017) Jun Xie et al.   MOLECULAR THERAPY

CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10

(2017) Guo-Xiang Ruan et al.   MOLECULAR THERAPY

Virus-Mediated Genome Editing via Homology-Directed Repair in Mitotic and Postmitotic Cells in Mammalian Brain

(2017) Jun Nishiyama et al.   NEURON

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

(2017) Eunji Kim et al.   Nature Communications

Hit and go CAS9 delivered through a lentiviral based self-limiting circuit

(2017) Gianluca Petris et al.   Nature Communications

CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice

(2017) Tsukasa Ohmori et al.   Scientific Reports

The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes

(2017) Nicolas Merienne et al.   Cell Reports

Lessons from Enzyme Kinetics Reveal Specificity Principles for RNA-Guided Nucleases in RNA Interference and CRISPR-Based Genome Editing

(2017) Namita Bisaria et al.   Cell Systems

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

(2016) Ciaran M Lee et al.   MOLECULAR THERAPY

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

In vivoblunt-end cloning through CRISPR/Cas9-facilitated non-homologous end-joining

(2016) Jonathan M. Geisinger et al.   NUCLEIC ACIDS RESEARCH

Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

(2016) Francis P. Pankowicz et al.   Nature Communications

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition

(2015) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

CRISPRseek: A Bioconductor Package to Identify Target-Specific Guide RNAs for CRISPR-Cas9 Genome-Editing Systems

(2014) Lihua J. Zhu et al.   PLoS One

Residual α-l-iduronidase activity in fibroblasts of mild to severe Mucopolysaccharidosis type I patients

(2013) Esmee Oussoren et al.   MOLECULAR GENETICS AND METABOLISM

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Efficient genome editing in zebrafish using a CRISPR-Cas system

(2013) Woong Y Hwang et al.   NATURE BIOTECHNOLOGY

RNA-guided editing of bacterial genomes using CRISPR-Cas systems

(2013) Wenyan Jiang et al.   NATURE BIOTECHNOLOGY

Orthogonal Cas9 proteins for RNA-guided gene regulation and editing

(2013) Kevin M Esvelt et al.   NATURE METHODS

Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis

(2013) Z. Hou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse

(2011) Dan Wang et al.   MOLECULAR GENETICS AND METABOLISM

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

(2011) Elitza Deltcheva et al.   NATURE

Integrative genomics viewer

(2011) James T Robinson et al.   NATURE BIOTECHNOLOGY

Tracking genome engineering outcome at individual DNA breakpoints

(2011) Michael T Certo et al.   NATURE METHODS

The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli

(2011) Rimantas Sapranauskas et al.   NUCLEIC ACIDS RESEARCH

BEDTools: a flexible suite of utilities for comparing genomic features

(2010) Aaron R. Quinlan et al.   BIOINFORMATICS

Search and clustering orders of magnitude faster than BLAST

(2010) Robert C. Edgar   BIOINFORMATICS

Galaxy: a comprehensive approach for supporting accessible, reproducible, and transparent computational research in the life sciences

(2010) Jeremy Goecks et al.   GENOME BIOLOGY

Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation

(2009) Dan Wang et al.   MOLECULAR GENETICS AND METABOLISM