CRISPR-derived genome editing therapies: Progress from bench to bedside

In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

(2021) Lukas Villiger et al.   Nature Biomedical Engineering

Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma

(2021) Nikhil C. Munshi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

(2021) John W Day et al.   LANCET NEUROLOGY

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

(2021) Mitchell L. Leibowitz et al.   NATURE GENETICS

Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit

(2021) Meaghan Van Alstyne et al.   NATURE NEUROSCIENCE

Rationally Designed Base Editors for Precise Editing of the Sickle Cell Disease Mutation

(2021) S. Haihua Chu et al.   CRISPR Journal

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

(2021) Gregory A. Newby et al.   NATURE

Detect-seq reveals out-of-protospacer editing and target-strand editing by cytosine base editors

(2021) Zhixin Lei et al.   NATURE METHODS

The current landscape of nucleic acid therapeutics

(2021) Jayesh A. Kulkarni et al.   Nature Nanotechnology

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

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

A brief history of human disease genetics

(2020) Melina Claussnitzer et al.   NATURE

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors

(2020) Jordan L. Doman et al.   NATURE BIOTECHNOLOGY

The GWAS Diversity Monitor tracks diversity by disease in real time

(2020) Melinda C. Mills et al.   NATURE GENETICS

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

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

Cas9 activates the p53 pathway and selects for p53-inactivating mutations

(2020) Oana M. Enache et al.   NATURE GENETICS

A rationally engineered cytosine base editor retains high on-target activity while reducing both DNA and RNA off-target effects

(2020) Erwei Zuo et al.   NATURE METHODS

KTE-X19 CAR T-Cell Therapy in Relapsed or Refractory Mantle-Cell Lymphoma

(2020) Michael Wang et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

(2020) Yi Yu et al.   Nature Communications

Recent Advances in CRISPR/Cas9 Delivery Strategies

(2020) Bon Ham Yip   Biomolecules

Whole-genome sequencing of patients with rare diseases in a national health system

(2020) Ernest Turro et al.   NATURE

Privacy challenges and research opportunities for genomic data sharing

(2020) Luca Bonomi et al.   NATURE GENETICS

Lisocabtagene maraleucel for patients with relapsed or refractory large B-cell lymphomas (TRANSCEND NHL 001): a multicentre seamless design study

(2020) Jeremy S Abramson et al.   LANCET

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

Viral Delivery Systems for CRISPR

(2019) Christine Xu et al.   Viruses-Basel

Genome-wide profiling of adenine base editor specificity by EndoV-seq

(2019) Puping Liang et al.   Nature Communications

HPLC methods for purity evaluation of man-made single-stranded RNAs

(2019) Anastassia Kanavarioti   Scientific Reports

CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations

(2019) Grégoire Cullot et al.   Nature Communications

Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos

(2019) Erwei Zuo et al.   SCIENCE

Cytosine, but not adenine, base editors induce genome-wide off-target mutations in rice

(2019) Shuai Jin et al.   SCIENCE

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

(2019) Piotr S. Kowalski et al.   MOLECULAR THERAPY

The Missing Diversity in Human Genetic Studies

(2019) Giorgio Sirugo et al.   CELL

Re-Engineering RNA Molecules into Therapeutic Agents

(2019) Martin Egli et al.   ACCOUNTS OF CHEMICAL RESEARCH

Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors

(2019) Julian Grünewald et al.   NATURE

Analysis and minimization of cellular RNA editing by DNA adenine base editors

(2019) Holly A. Rees et al.   Science Advances

Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis

(2019) Changyang Zhou et al.   NATURE

An artificial triazole backbone linkage provides a split-and-click strategy to bioactive chemically modified CRISPR sgRNA

(2019) Lapatrada Taemaitree et al.   Nature Communications

CRISPR DNA base editors with reduced RNA off-target and self-editing activities

(2019) Julian Grünewald et al.   NATURE BIOTECHNOLOGY

Adenine base editors catalyze cytosine conversions in human cells

(2019) Heon Seok Kim et al.   NATURE BIOTECHNOLOGY

CRISPRitz: rapid, high-throughput and variant-aware in silico off-target site identification for CRISPR genome editing

(2019) Samuele Cancellieri et al.   BIOINFORMATICS

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

(2019) Andrew V. Anzalone et al.   NATURE

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

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

Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors

(2019) Beau R. Webber et al.   Nature Communications

Gene therapy for sickle cell disease: An update

(2018) Selami Demirci et al.   CYTOTHERAPY

Precise and Predictable CRISPR Chromosomal Rearrangements Reveal Principles of Cas9-Mediated Nucleotide Insertion

(2018) Jia Shou et al.   MOLECULAR CELL

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

(2018) Shannon L. Maude et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR/Cas9 cleavages in budding yeast reveal templated insertions and strand-specific insertion/deletion profiles

(2018) Brenda R. Lemos et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In vivo base editing of post-mitotic sensory cells

(2018) Wei-Hsi Yeh et al.   Nature Communications

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

(2018) Sriram Vaidyanathan et al.   Molecular Therapy-Nucleic Acids

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Target-Specific Precision of CRISPR-Mediated Genome Editing

(2018) Anob M. Chakrabarti et al.   MOLECULAR CELL

Predictable and precise template-free CRISPR editing of pathogenic variants

(2018) Max W. Shen et al.   NATURE

Predicting the mutations generated by repair of Cas9-induced double-strand breaks

(2018) Felicity Allen et al.   NATURE BIOTECHNOLOGY

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

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

Tisagenlecleucel in Adult Relapsed or Refractory Diffuse Large B-Cell Lymphoma

(2018) Stephen J. Schuster et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

(2017) Y Bill Kim et al.   NATURE BIOTECHNOLOGY

Genome-wide target specificities of CRISPR RNA-guided programmable deaminases

(2017) Daesik Kim et al.   NATURE BIOTECHNOLOGY

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma

(2017) Sattva S. Neelapu et al.   NEW ENGLAND JOURNAL OF MEDICINE

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery

(2017) Holly A. Rees et al.   Nature Communications

Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity

(2017) Alexis C. Komor et al.   Science Advances

Addressing the challenge of high-priced prescription drugs in the era of precision medicine: A systematic review of drug life cycles, therapeutic drug markets and regulatory frameworks

(2017) Toon van der Gronde et al.   PLoS One

Conjugation and Evaluation of Triazole-Linked Single Guide RNA for CRISPR-Cas9 Gene Editing

(2016) Kaizhang He et al.   CHEMBIOCHEM

Genomics is failing on diversity

(2016) Alice B. Popejoy et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Enabling functional genomics with genome engineering

(2015) Isaac B. Hilton et al.   GENOME RESEARCH

N 1 -methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice

(2015) Oliwia Andries et al.   JOURNAL OF CONTROLLED RELEASE

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Toll-like receptor 8 senses degradation products of single-stranded RNA

(2015) Hiromi Tanji et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Toll-like receptor 8 senses degradation products of single-stranded RNA

(2015) Hiromi Tanji et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

CCTop: An Intuitive, Flexible and Reliable CRISPR/Cas9 Target Prediction Tool

(2015) Manuel Stemmer et al.   PLoS One

Transcriptome-wide Mapping Reveals Widespread Dynamic-Regulated Pseudouridylation of ncRNA and mRNA

(2014) Schraga Schwartz et al.   CELL

Guide RNA Functional Modules Direct Cas9 Activity and Orthogonality

(2014) Alexandra E. Briner et al.   MOLECULAR CELL

Pseudouridine profiling reveals regulated mRNA pseudouridylation in yeast and human cells

(2014) Thomas M. Carlile et al.   NATURE

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

Eukaryotic translation initiation is controlled by cooperativity effects within ternary complexes of 4E-BP1, eIF4E, and the mRNA 5′ cap

(2013) Anna Modrak-Wojcik et al.   FEBS LETTERS

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

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

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

crRNA and tracrRNA guide Cas9-mediated DNA interference inStreptococcus thermophilus

(2013) Tautvydas Karvelis et al.   RNA Biology

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

Quality Aspects of Oligonucleotide Drug Development: Specifications for Active Pharmaceutical Ingredients

(2012) Daniel Capaldi et al.   DRUG INFORMATION JOURNAL

Nonviral delivery of self-amplifying RNA vaccines

(2012) A. J. Geall 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

Streamlined Process for the Chemical Synthesis of RNA Using 2′-O-Thionocarbamate-Protected Nucleoside Phosphoramidites in the Solid Phase

(2011) Douglas J. Dellinger et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

(2011) Elitza Deltcheva et al.   NATURE

Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

(2011) Katalin Karikó et al.   NUCLEIC ACIDS RESEARCH

Nucleoside modifications in RNA limit activation of 2'-5'-oligoadenylate synthetase and increase resistance to cleavage by RNase L

(2011) B. R. Anderson et al.   NUCLEIC ACIDS RESEARCH

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation

(2010) Bart R. Anderson et al.   NUCLEIC ACIDS RESEARCH

Regulation of mRNA decapping

(2010) You Li et al.   Wiley Interdisciplinary Reviews-RNA

DNA Repair in Mammalian Cells

(2009) C. Kunz et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

(2008) Katalin Karikó et al.   MOLECULAR THERAPY