What did CRISPR-Cas9 accomplish in its first 10 years?

Off-target effects in CRISPR/Cas9 gene editing

(2023) Congting Guo et al.   Frontiers in Bioengineering and Biotechnology

CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease In Vivo

(2022) Hyung Ho Yoon et al.   CRISPR Journal

Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy

(2022) Mohammed Fatih Rasul et al.   Molecular Cancer

CRISPR-Cas9 induces large structural variants at on-target and off-target sites in vivo that segregate across generations

(2022) Ida Höijer et al.   Nature Communications

CRISPR-Cas9 treatment partially restores amyloid-β 42/40 in human fibroblasts with the Alzheimer’s disease PSEN1 M146L mutation

(2022) Evangelos Konstantinidis et al.   Molecular Therapy-Nucleic Acids

Efficacy and Safety of an Investigational Single-course CRISPR Base Editing Therapy Targeting PCSK9 in Non-human Primate and Mouse Models

(2022) Richard G. Lee et al.   CIRCULATION

Phase 1 clinical trial of CRISPR-engineered CAR19 universal T cells for treatment of children with refractory B cell leukemia

(2022) Giorgio Ottaviano et al.   Science Translational Medicine

CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro

(2022) Katharina E. Meijboom et al.   Nature Communications

Non-viral precision T cell receptor replacement for personalized cell therapy

(2022) Susan P. Foy et al.   NATURE

CRISPR technologies and the search for the PAM-free nuclease

(2021) Daphne Collias et al.   Nature Communications

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9

(2021) Lili Wang et al.   MOLECULAR THERAPY

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus

(2021) Sriram Vaidyanathan et al.   MOLECULAR THERAPY

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

Nanoparticle Delivery of CRISPR/Cas9 for Genome Editing

(2021) Li Duan et al.   Frontiers in Genetics

Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing

(2021) Jessica D. Gumerson et al.   GENE THERAPY

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

In vitro genome editing rescues parkinsonism phenotypes in induced pluripotent stem cells-derived dopaminergic neurons carrying LRRK2 p.G2019S mutation

(2021) Kuo-Hsuan Chang et al.   Stem Cell Research & Therapy

Evaluating CRISPR-based prime editing for cancer modeling and CFTR repair in organoids

(2021) Maarten H Geurts et al.   Life Science Alliance

A systematic genome-wide mapping of oncogenic mutation selection during CRISPR-Cas9 genome editing

(2021) Sanju Sinha et al.   Nature Communications

Suppression of unwanted CRISPR-Cas9 editing by co-administration of catalytically inactivating truncated guide RNAs

(2020) John C. Rose et al.   Nature Communications

CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future

(2020) Fathema Uddin et al.   Frontiers in Oncology

Application of different types of CRISPR/Cas-based systems in bacteria

(2020) Zhenquan Liu et al.   Microbial Cell Factories

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

(2020) Erica B. Esrick et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

Guidelines for optimized gene knockout using CRISPR/Cas9

(2019) Claude Van Campenhout et al.   BIOTECHNIQUES

HIV-specific humoral immune responses by CRISPR/Cas9-edited B cells

(2019) Harald Hartweger et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Life-Long AAV-Mediated CRISPR Genome Editing in Dystrophic Heart Improves Cardiomyopathy without Causing Serious Lesions in mdx Mice

(2019) Li Xu et al.   MOLECULAR THERAPY

Sequential LASER ART and CRISPR Treatments Eliminate HIV-1 in a Subset of Infected Humanized Mice

(2019) Prasanta K. Dash et al.   Nature Communications

CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington’s disease mouse model

(2019) Freja K. Ekman et al.   Molecular Therapy-Nucleic Acids

In Vivo AAV-CRISPR/Cas9-Mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia

(2019) Huan Zhao et al.   CIRCULATION

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

(2019) Hainan Chen et al.   Scientific Reports

Recent advances in CRISPR/Cas9-mediated knock-ins in mammalian cells

(2019) Mehdi Banan   JOURNAL OF BIOTECHNOLOGY

Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice

(2018) Shen Shen et al.   HUMAN GENE THERAPY

In vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency

(2018) Chun-Qing Song et al.   HUMAN GENE THERAPY

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

CRISPR/Cas9 system targeting regulatory genes of HIV-1 inhibits viral replication in infected T-cell cultures

(2018) Youdiil Ophinni et al.   Scientific Reports

Inherited Retinal Degenerations: Current Landscape and Knowledge Gaps

(2018) Jacque L. Duncan et al.   Translational Vision Science & Technology

Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates α1-antitrypsin Deficiency Phenotype

(2018) Mikael Bjursell et al.   EBioMedicine

In utero CRISPR-mediated therapeutic editing of metabolic genes

(2018) Avery C. Rossidis et al.   NATURE MEDICINE

Global birth prevalence and mortality from inborn errors of metabolism: a systematic analysis of the evidence

(2018) Donald Waters et al.   Journal of Global Health

Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration

(2017) Erin R. Burnight 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

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs

(2017) Lixia Wang et al.   Protein & Cell

In vivo genome editing improves motor function and extends survival in a mouse model of ALS

(2017) Thomas Gaj et al.   Science Advances

CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia

(2017) Alexandra C. Chadwick et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Origins of Programmable Nucleases for Genome Engineering

(2016) Srinivasan Chandrasegaran et al.   JOURNAL OF MOLECULAR BIOLOGY

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

(2016) Megan D Hoban et al.   MOLECULAR THERAPY

CRISPR-Cas: biology, mechanisms and relevance

(2016) Frank Hille et al.   PHILOSOPHICAL TRANSACTIONS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES

Advances in therapeutic CRISPR/Cas9 genome editing

(2016) Nataša Savić et al.   Translational Research

Systematic quantification of HDR and NHEJ reveals effects of locus, nuclease, and cell type on genome-editing

(2016) Yuichiro Miyaoka et al.   Scientific Reports

CRISPR-Cas9 mediated efficient PD-1 disruption on human primary T cells from cancer patients

(2016) Shu Su et al.   Scientific Reports

The therapeutic application of CRISPR/Cas9 technologies for HIV

(2015) Sheena Saayman et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

(2015) Xiao-Hui Zhang et al.   Molecular Therapy-Nucleic Acids

Expanding the genetic editing tool kit: ZFNs, TALENs, and CRISPR-Cas9

(2014) Rajat M. Gupta et al.   JOURNAL OF CLINICAL INVESTIGATION

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

(2014) Bin Shen et al.   NATURE METHODS

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

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

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 Mechanism of Double-Strand DNA Break Repair by the Nonhomologous DNA End-Joining Pathway

(2010) Michael R. Lieber   Annual Review of Biochemistry