Small Nucleic Acids and the Path to the Clinic for Anti-CRISPR

N-of-1 drugs push biopharma frontiers

(2020) Asher Mullard   NATURE REVIEWS DRUG DISCOVERY

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

(2020) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

A Small Molecule-Controlled Cas9 Repressible System

(2020) Youjun Wu et al.   Molecular Therapy-Nucleic Acids

Coupling Cas9 to artificial inhibitory domains enhances CRISPR-Cas9 target specificity

(2020) Sabine Aschenbrenner et al.   Science Advances

Potent CRISPR-Cas9 inhibitors fromStaphylococcusgenomes

(2020) Kyle E. Watters et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Small-Molecule Control of Super-Mendelian Inheritance in Gene Drives

(2020) Víctor López Del Amo et al.   Cell Reports

Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing

(2020) Muhammad Naeem et al.   Cells

A cell cycle-dependent CRISPR-Cas9 activation system based on an anti-CRISPR protein shows improved genome editing accuracy

(2020) Daisuke Matsumoto et al.   Communications Biology

CRISPR-cas gene-editing as plausible treatment of neuromuscular and nucleotide-repeat-expansion diseases: A systematic review

(2019) Haris Babačić et al.   PLoS One

Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates

(2019) Maja M Janas et al.   NUCLEIC ACIDS RESEARCH

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

A microRNA-inducible CRISPR–Cas9 platform serves as a microRNA sensor and cell-type-specific genome regulation tool

(2019) Xi-Wen Wang et al.   NATURE CELL BIOLOGY

CRISPR/Cas9 technology as a potent molecular tool for gene therapy

(2019) Ansar Karimian et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Gymnotic Delivery of LNA Mixmers Targeting Viral SREs Induces HIV-1 mRNA Degradation

(2019) Frank Hillebrand et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

A High-Throughput Platform to Identify Small-Molecule Inhibitors of CRISPR-Cas9

(2019) Basudeb Maji et al.   CELL

Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

(2019) Panayiota Papasavva et al.   Molecular Diagnosis & Therapy

Broad-spectrum enzymatic inhibition of CRISPR-Cas12a

(2019) Gavin J. Knott et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

An anti-CRISPR protein disables type V Cas12a by acetylation

(2019) Liyong Dong et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Tissue-specific (ts)CRISPR as an efficient strategy for in vivo screening in Drosophila

(2019) Hagar Meltzer et al.   Nature Communications

Rationally Designed Small Nucleic Acid-Based Inhibitors of CRISPR-Cas9

(2019) Christopher L. Barkau et al.   Nucleic Acid Therapeutics

Steric Inhibition of 5′ UTR Regulatory Elements Results in Upregulation of Human CFTR

(2019) Shruti Sasaki et al.   MOLECULAR THERAPY

Anti‐CRISPR AcrIIC3 discriminates between Cas9 orthologs via targeting the variable surface of the HNH nuclease domain

(2019) Youngim Kim et al.   FEBS Journal

CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

(2019) Lei Xu et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2019) Jooyoung Lee et al.   RNA

Dual Mechanisms of Action of Self-Delivering, Anti-HIV-1 FANA Oligonucleotides as a Potential New Approach to HIV Therapy

(2019) Mayumi Takahashi et al.   Molecular Therapy-Nucleic Acids

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

(2019) Jinkuk Kim et al.   NEW ENGLAND JOURNAL OF MEDICINE

AcrIIA5 Inhibits a Broad Range of Cas9 Orthologs by Preventing DNA Target Cleavage

(2019) Guoxu Song et al.   Cell Reports

Anti-CRISPR AcrIIA5 Potently Inhibits All Cas9 Homologs Used for Genome Editing

(2019) Bianca Garcia et al.   Cell Reports

Oligonucleotides to the (Gene) Rescue: FDA Approvals 2017–2019

(2019) Jacqueline Rüger et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Versatile High-Throughput Fluorescence Assay for Monitoring Cas9 Activity

(2018) Kyle J. Seamon et al.   ANALYTICAL CHEMISTRY

CRISPR base editors: genome editing without double-stranded breaks

(2018) Ayman Eid et al.   BIOCHEMICAL JOURNAL

Chimeric Guides Probe and Enhance Cas9 Biochemical Activity

(2018) Zachary J. Kartje et al.   BIOCHEMISTRY

Harnessing CRISPR/Cas systems for programmable transcriptional and post-transcriptional regulation

(2018) Ahmed Mahas et al.   BIOTECHNOLOGY ADVANCES

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

Drug metabolism and pharmacokinetic strategies for oligonucleotide- and mRNA-based drug development

(2018) Shalini Andersson et al.   DRUG DISCOVERY TODAY

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Antibody-Antisense Oligonucleotide Conjugate Downregulates a Key Gene in Glioblastoma Stem Cells

(2018) Amy E. Arnold et al.   Molecular Therapy-Nucleic Acids

Strategies for controlling CRISPR/Cas9 off-target effects and biological variations in mammalian genome editing experiments

(2018) Michelle L. Kimberland et al.   JOURNAL OF BIOTECHNOLOGY

OUP accepted manuscript

(2018)   NUCLEIC ACIDS RESEARCH

Discovery of widespread Type I and Type V CRISPR-Cas inhibitors

(2018) Nicole D. Marino et al.   SCIENCE

Systematic discovery of natural CRISPR-Cas12a inhibitors

(2018) Kyle E. Watters et al.   SCIENCE

CRISPR-Based Technologies: Impact of RNA-Targeting Systems

(2018) Michael P. Terns   MOLECULAR CELL

Synthetic switch to minimize CRISPR off-target effects by self-restricting Cas9 transcription and translation

(2018) Chih-Che Shen et al.   NUCLEIC ACIDS RESEARCH

Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways

(2018) Maire F Osborn et al.   NUCLEIC ACIDS RESEARCH

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo

(2018) Annabelle Biscans et al.   NUCLEIC ACIDS RESEARCH

Extensive CRISPR RNA modification reveals chemical compatibility and structure-activity relationships for Cas9 biochemical activity

(2018) Daniel O’Reilly et al.   NUCLEIC ACIDS RESEARCH

Structural basis of CRISPR–SpyCas9 inhibition by an anti-CRISPR protein

(2017) De Dong et al.   NATURE

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Towards CRISPR/Cas crops - bringing together genomics and genome editing

(2017) Armin Scheben et al.   NEW PHYTOLOGIST

Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

(2017) Michael Tanowitz et al.   NUCLEIC ACIDS RESEARCH

An anti-CRISPR from a virulent streptococcal phage inhibits Streptococcus pyogenes Cas9

(2017) Alexander P. Hynes et al.   Nature Microbiology

Disabling Cas9 by an anti-CRISPR DNA mimic

(2017) Jiyung Shin et al.   Science Advances

Naturally Occurring Off-Switches for CRISPR-Cas9

(2016) April Pawluk et al.   CELL

Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles

(2016) Tsukasa Sugo et al.   JOURNAL OF CONTROLLED RELEASE

A Self-restricted CRISPR System to Reduce Off-target Effects

(2016) Yanhao Chen et al.   MOLECULAR THERAPY

CRISPR gene-editing tested in a person for the first time

(2016) David Cyranoski   NATURE

Structural basis of Cas3 inhibition by the bacteriophage protein AcrF3

(2016) Xiaofei Wang et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector

(2016) Omar O. Abudayyeh et al.   SCIENCE

Structural basis of Cas3 inhibition by the bacteriophage protein AcrF3

(2016) Xiaofei Wang et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Multiple mechanisms for CRISPR–Cas inhibition by anti-CRISPR proteins

(2015) Joseph Bondy-Denomy et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

A split-Cas9 architecture for inducible genome editing and transcription modulation

(2015) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

A CRISPR-Cas9 gene drive system targeting female reproduction in the malaria mosquito vector Anopheles gambiae

(2015) Andrew Hammond et al.   NATURE BIOTECHNOLOGY

Small molecule–triggered Cas9 protein with improved genome-editing specificity

(2015) Kevin M Davis et al.   Nature Chemical Biology

Identification and characterization of intracellular proteins that bind oligonucleotides with phosphorothioate linkages

(2015) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

Opinion: Is CRISPR-based gene drive a biocontrol silver bullet or global conservation threat?: Fig. 1.

(2015) Bruce L. Webber et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Highly efficient Cas9-mediated gene drive for population modification of the malaria vector mosquitoAnopheles stephensi

(2015) Valentino M. Gantz et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA

(2014) S. Ramakrishna et al.   GENOME RESEARCH

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Microhomology-based choice of Cas9 nuclease target sites

(2014) Sangsu Bae et al.   NATURE METHODS

Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB–siRNA conjugates

(2014) Trinna L. Cuellar et al.   NUCLEIC ACIDS RESEARCH

Antisense Oligonucleotides Capable of Promoting Specific Target mRNA Reduction via Competing RNase H1-Dependent and Independent Mechanisms

(2014) Timothy A. Vickers et al.   PLoS One

A New Group of Phage Anti-CRISPR Genes Inhibits the Type I-E CRISPR-Cas System of Pseudomonas aeruginosa

(2014) A. Pawluk et al.   mBio

Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9

(2014) Shota Nakade et al.   Nature Communications

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

Targeting Protein-Protein Interaction by Small Molecules

(2013) Lingyan Jin et al.   Annual Review of Pharmacology and Toxicology

Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression

(2013) Lei S. Qi et al.   CELL

Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides

(2013) Mehrdad Dirin et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

RNA-Guided Genome Editing in Plants Using a CRISPR–Cas System

(2013) Kabin Xie et al.   Molecular Plant

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

A Chemical View of Oligonucleotides for Exon Skipping and Related Drug Applications

(2013) Peter Järver et al.   Nucleic Acid Therapeutics

Bacteriophage genes that inactivate the CRISPR/Cas bacterial immune system

(2012) Joe Bondy-Denomy et al.   NATURE

The solution structure of double helical arabino nucleic acids (ANA and 2′F-ANA): effect of arabinoses in duplex-hairpin interconversion

(2012) Nerea Martín-Pintado et al.   NUCLEIC ACIDS RESEARCH

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

Targeted Delivery of PLK1-siRNA by ScFv Suppresses Her2+ Breast Cancer Growth and Metastasis

(2012) Y.-d. Yao et al.   Science Translational Medicine

Antisense 2′-Deoxy, 2′-Fluoroarabino Nucleic Acid (2′F-ANA) Oligonucleotides: In Vitro Gymnotic Silencers of Gene Expression Whose Potency Is Enhanced by Fatty Acids

(2012) Naira Souleimanian et al.   Molecular Therapy-Nucleic Acids

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents

(2009) C. A. Stein et al.   NUCLEIC ACIDS RESEARCH

T Cell-Specific siRNA Delivery Suppresses HIV-1 Infection in Humanized Mice

(2008) Priti Kumar et al.   CELL

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Delivery Vehicles for Small Interfering RNA In Vivo

(2008) Antonin R. de Fougerolles   HUMAN GENE THERAPY

Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis

(2008) Md Rowshon Alam et al.   NUCLEIC ACIDS RESEARCH