Delivery technologies for genome editing

CRISPR-Cas9 Targeting ofPCSK9in Human Hepatocytes In Vivo—Brief ReportSignificance

(2016) Xiao Wang et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Optimizing T-cell receptor gene therapy for hematologic malignancies

(2016) Emma C. Morris et al.   BLOOD

RNAi-nanoparticulate manipulation of gene expression as a new functional genomics tool in the liver

(2016) Hao Yin et al.   JOURNAL OF HEPATOLOGY

Cystic fibrosis

(2016) J Stuart Elborn   LANCET

573. Vector Free Genome Editing of Immune Cells for Cell Therapy

(2016) Tia DiTommaso et al.   MOLECULAR THERAPY

Engineered Viruses as Genome Editing Devices

(2016) Xiaoyu Chen et al.   MOLECULAR THERAPY

Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

(2016) Thomas Gaj et al.   MOLECULAR THERAPY

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

(2016) Li Xu et al.   MOLECULAR THERAPY

CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa

(2016) Wen-Hsuan Wu et al.   MOLECULAR THERAPY

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

(2016) Suk See De Ravin et al.   NATURE BIOTECHNOLOGY

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

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

The future of cancer treatment: immunomodulation, CARs and combination immunotherapy

(2016) Danny N. Khalil et al.   Nature Reviews Clinical Oncology

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Multi-reporter selection for the design of active and more specific zinc-finger nucleases for genome editing

(2016) Benjamin L. Oakes et al.   Nature Communications

Targeted approaches to induce immune tolerance for Pompe disease therapy

(2016) Phillip A Doerfler et al.   Molecular Therapy-Methods & Clinical Development

Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing

(2015) Wujin Sun et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

(2015) R. Sharma et al.   BLOOD

Efficient Intracellular Delivery of Native Proteins

(2015) Diego S. D’Astolfo et al.   CELL

Adenovirus-Mediated Somatic Genome Editing ofPtenby CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

(2015) Dan Wang et al.   HUMAN GENE THERAPY

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

Hemophilia Gene Therapy: Caught Between a Cure and an Immune Response

(2015) Roland W. Herzog   MOLECULAR THERAPY

A New Era of Innovation for CAR T-cell Therapy

(2015) Robert M Frederickson   MOLECULAR THERAPY

Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma

(2015) Joal D Beane et al.   MOLECULAR THERAPY

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors

(2015) Xiaoling Wang et al.   NATURE BIOTECHNOLOGY

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

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

(2015) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

DNA-binding-domain fusions enhance the targeting range and precision of Cas9

(2015) Mehmet Fatih Bolukbasi et al.   NATURE METHODS

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

(2015) Daesik Kim et al.   NATURE METHODS

Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery

(2015) Jianbin Wang et al.   NUCLEIC ACIDS RESEARCH

Generation of knock-in primary human T cells using Cas9 ribonucleoproteins

(2015) Kathrin Schumann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Rational design of a split-Cas9 enzyme complex

(2015) Addison V. Wright et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A prudent path forward for genomic engineering and germline gene modification

(2015) D. Baltimore et al.   SCIENCE

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Genome-wide inactivation of porcine endogenous retroviruses (PERVs)

(2015) L. Yang et al.   SCIENCE

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

(2015) Blythe D. Sather et al.   Science Translational Medicine

CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes

(2015) Puping Liang et al.   Protein & Cell

Virus-Based Nanoparticles as Versatile Nanomachines

(2015) Kristopher J. Koudelka et al.   Annual Review of Virology

CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation

(2015) X. Han et al.   Science Advances

CRISPR/Cas9-mediated genome engineering: An adeno-associated viral (AAV) vector toolbox

(2014) Elena Senís et al.   Biotechnology Journal

Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9

(2014) Pankaj K. Mandal et al.   Cell Stem Cell

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

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

Current status of haemophilia gene therapy

(2014) K. H. High et al.   HAEMOPHILIA

Recent developments in the treatment of age-related macular degeneration

(2014) Frank G. Holz et al.   JOURNAL OF CLINICAL INVESTIGATION

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

CRISPR-mediated direct mutation of cancer genes in the mouse liver

(2014) Wen Xue et al.   NATURE

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

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

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification

(2014) John P Guilinger 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

Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases

(2014) Richard L Frock et al.   NATURE BIOTECHNOLOGY

Adenoviral vector DNA for accurate genome editing with engineered nucleases

(2014) Maarten Holkers et al.   NATURE METHODS

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cell-Penetrating Peptide-Mediated Delivery of TALEN Proteins via Bioconjugation for Genome Engineering

(2014) Jia Liu et al.   PLoS One

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

(2014) C. J. Braun et al.   Science Translational Medicine

Robust ZFN-mediated genome editing in adult hemophilic mice

(2013) X. M. Anguela et al.   BLOOD

Toward eliminating HLA class I expression to generate universal cells from allogeneic donors

(2013) H. Torikai et al.   BLOOD

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering

(2013) Hui Yang et al.   CELL

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

A library of TAL effector nucleases spanning the human genome

(2013) Yongsub Kim et al.   NATURE BIOTECHNOLOGY

Rare-disease genetics in the era of next-generation sequencing: discovery to translation

(2013) Kym M. Boycott et al.   NATURE REVIEWS GENETICS

In silico abstraction of zinc finger nuclease cleavage profiles reveals an expanded landscape of off-target sites

(2013) Jeffry D. Sander et al.   NUCLEIC ACIDS RESEARCH

A vector-free microfluidic platform for intracellular delivery

(2013) A. Sharei 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

Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?

(2013) J. A. Gorzelany et al.   Science Translational Medicine

Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification

(2013) Kimberly A Wilson et al.   Molecular Therapy-Nucleic Acids

Highly active zinc-finger nucleases by extended modular assembly

(2012) M. S. Bhakta et al.   GENOME RESEARCH

Playing the End Game: DNA Double-Strand Break Repair Pathway Choice

(2012) J. Ross Chapman et al.   MOLECULAR CELL

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

(2012) Christian Mueller et al.   MOLECULAR THERAPY

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

(2012) Elena Provasi et al.   NATURE MEDICINE

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins

(2012) Thomas Gaj et al.   NATURE METHODS

Therapeutic opportunities for manipulating TReg cells in autoimmunity and cancer

(2012) Harald von Boehmer et al.   NATURE REVIEWS DRUG DISCOVERY

TALENs: a widely applicable technology for targeted genome editing

(2012) J. Keith Joung et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

AAV Vector Biology in Primates: Finding the Missing Link?

(2011) Roland W Herzog et al.   MOLECULAR THERAPY

A Cell-penetrating Peptide Suppresses Inflammation by Inhibiting NF-κB Signaling

(2011) Yu Fu Wang et al.   MOLECULAR THERAPY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

An unbiased genome-wide analysis of zinc-finger nuclease specificity

(2011) Richard Gabriel et al.   NATURE BIOTECHNOLOGY

Chemically defined conditions for human iPSC derivation and culture

(2011) Guokai Chen et al.   NATURE METHODS

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Homing Endonucleases: From Microbial Genetic Invaders to Reagents for Targeted DNA Modification

(2011) Barry L. Stoddard   STRUCTURE

Regulation of Homologous Recombination in Eukaryotes

(2010) Wolf-Dietrich Heyer et al.   Annual Review of Genetics

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

(2010) R. C. DeKelver et al.   GENOME RESEARCH

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion

(2010) Cem Şöllü et al.   NUCLEIC ACIDS RESEARCH

Precise genome modification in the crop species Zea mays using zinc-finger nucleases

(2009) Vipula K. Shukla et al.   NATURE

The promises and pitfalls of RNA-interference-based therapeutics

(2009) Daniela Castanotto et al.   NATURE

The origins of the identification and isolation of hematopoietic stem cells, and their capability to induce donor-specific transplantation tolerance and treat autoimmune diseases

(2008) I. L. Weissman et al.   BLOOD

Viral vectors: from virology to transgene expression

(2008) D Bouard et al.   BRITISH JOURNAL OF PHARMACOLOGY

Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

(2008) S E Khorsandi et al.   CANCER GENE THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY