Therapeutic in vivo delivery of gene editing agents

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

(2022) Samagya Banskota et al.   CELL

Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

(2022) Eman A. Taha et al.   JOURNAL OF CONTROLLED RELEASE

CRISPR-based genome editing through the lens of DNA repair

(2022) Tarun S. Nambiar et al.   MOLECULAR CELL

A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver

(2022) Chunwei Zheng et al.   MOLECULAR THERAPY

CAR T cells produced in vivo to treat cardiac injury

(2022) Joel G. Rurik et al.   SCIENCE

Nuclear and mitochondrial DNA editing in human cells with zinc finger deaminases

(2022) Kayeong Lim et al.   Nature Communications

In vivo mitochondrial base editing via adeno-associated viral delivery to mouse post-mitotic tissue

(2022) Pedro Silva-Pinheiro et al.   Nature Communications

Targeted A-to-G base editing in human mitochondrial DNA with programmable deaminases

(2022) Sung-Ik Cho et al.   CELL

A split prime editor with untethered reverse transcriptase and circular RNA template

(2022) Bin Liu et al.   NATURE BIOTECHNOLOGY

In vivo prime editing of a metabolic liver disease in mice

(2022) Desirée Böck et al.   Science Translational Medicine

In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice

(2021) Luke W. Koblan et al.   NATURE

Targeting herpes simplex virus with CRISPR–Cas9 cures herpetic stromal keratitis in mice

(2021) Di Yin et al.   NATURE BIOTECHNOLOGY

The clinical landscape for AAV gene therapies

(2021) Dmitry A. Kuzmin et al.   NATURE REVIEWS DRUG DISCOVERY

Efficient and high-fidelity base editor with expanded PAM compatibility for cytidine dinucleotide

(2021) Zhiquan Liu et al.   Science China-Life Sciences

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

(2021) Lukas Villiger et al.   Nature Biomedical Engineering

Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice

(2021) Sikai Ling et al.   Nature Biomedical Engineering

Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq

(2021) Giandomenico Turchiano et al.   Cell Stem Cell

Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?

(2021) Dimitrios Laurin Wagner et al.   GENE THERAPY

Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

(2021) Min Qiu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mitochondrial DNA editing in mice with DddA-TALE fusion deaminases

(2021) Hyunji Lee et al.   Nature Communications

Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair proteins

(2021) Liwei Chen et al.   Nature Communications

Adenine Base Editor Ribonucleoproteins Delivered by Lentivirus-Like Particles Show High On-Target Base Editing and Undetectable RNA Off-Target Activities

(2021) Pin Lyu et al.   CRISPR Journal

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

(2021) Chang Li et al.   Blood Advances

mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability

(2021) Linde Schoenmaker et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

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

(2021) Kiran Musunuru et al.   NATURE

The NIH Somatic Cell Genome Editing program

(2021) Krishanu Saha et al.   NATURE

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

(2021) Tanja Rothgangl et al.   NATURE BIOTECHNOLOGY

Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations

(2021) Lijie Wang et al.   NATURE CELL BIOLOGY

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

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

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

(2021) Pengpeng Liu et al.   Nature Communications

Anti-AAV Antibodies in AAV Gene Therapy: Current Challenges and Possible Solutions

(2021) Thomas Weber   Frontiers in Immunology

Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

(2021) F. Chemello et al.   Science Advances

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

(2021) Mohammadsharif Tabebordbar et al.   CELL

In vivo somatic cell base editing and prime editing

(2021) Gregory A. Newby et al.   MOLECULAR THERAPY

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

(2021) Gregory A. Newby et al.   NATURE

Efficient C•G-to-G•C base editors developed using CRISPRi screens, target-library analysis, and machine learning

(2021) Luke W. Koblan et al.   NATURE BIOTECHNOLOGY

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

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

Mammalian retrovirus-like protein PEG10 packages its own mRNA and can be pseudotyped for mRNA delivery

(2021) Michael Segel et al.   SCIENCE

Efficient precise in vivo base editing in adult dystrophic mice

(2021) Li Xu et al.   Nature Communications

Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering

(2021) Jennifer R. Hamilton et al.   Cell Reports

The impact of lentiviral vector genome size and producer cell genomic to gag-pol mRNA ratios on packaging efficiency and titre

(2021) Nathan P. Sweeney et al.   Molecular Therapy-Methods & Clinical Development

High-purity production and precise editing of DNA base editing ribonucleoproteins

(2021) Hyeon-Ki Jang et al.   Science Advances

Enhanced prime editing systems by manipulating cellular determinants of editing outcomes

(2021) Peter J. Chen et al.   CELL

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

(2021) Holly A. Rees et al.   MOLECULAR THERAPY

Engineered pegRNAs improve prime editing efficiency

(2021) James W. Nelson et al.   NATURE BIOTECHNOLOGY

AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset

(2021) David Goertsen et al.   NATURE NEUROSCIENCE

On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles

(2021) Sean A. Dilliard et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

(2021) Eriya Kenjo et al.   Nature Communications

Comprehensive deletion landscape of CRISPR-Cas9 identifies minimal RNA-guided DNA-binding modules

(2021) Arik Shams et al.   Nature Communications

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

(2021) Raed Ibraheim et al.   Nature Communications

DdCBE mediates efficient and inheritable modifications in mouse mitochondrial genome

(2021) Jiayin Guo et al.   Molecular Therapy-Nucleic Acids

Can Adeno-Associated Viral Vectors Deliver Effectively Large Genes?

(2020) Patrizia Tornabene et al.   HUMAN GENE THERAPY

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

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

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

The promise and challenge of therapeutic genome editing

(2020) Jennifer A. Doudna   NATURE

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

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

Programmable base editing of mutated TERT promoter inhibits brain tumour growth

(2020) Xinjian Li et al.   NATURE CELL BIOLOGY

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

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

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

(2020) Dan Wang et al.   CELL

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

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

Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

(2020) Michelle F. Richter et al.   NATURE BIOTECHNOLOGY

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

(2020) Qiang Cheng et al.   Nature Nanotechnology

Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope

(2020) Tingting Jiang et al.   Nature Communications

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

(2020) Peter Gee et al.   Nature Communications

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

(2020) Ziying Hu et al.   PLOS BIOLOGY

Delivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editing

(2020) Tuo Wei et al.   ACS Nano

Lipid nanoparticle technology for therapeutic gene regulation in the liver

(2020) Dominik Witzigmann et al.   ADVANCED DRUG DELIVERY REVIEWS

A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing

(2020) Beverly Y. Mok et al.   NATURE

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

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

CRISPR C-to-G base editors for inducing targeted DNA transversions in human cells

(2020) Ibrahim C. Kurt et al.   NATURE BIOTECHNOLOGY

The delivery challenge: fulfilling the promise of therapeutic genome editing

(2020) Joost van Haasteren et al.   NATURE BIOTECHNOLOGY

Highly efficient ‘hit-and-run’ genome editing with unconcentrated lentivectors carrying Vpr.Prot.Cas9 protein produced from RRE-containing transcripts

(2020) Ivana Indikova et al.   NUCLEIC ACIDS RESEARCH

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

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

(2020) Tuo Wei et al.   Nature Communications

In vivo–directed evolution of adeno-associated virus in the primate retina

(2020) Leah C. Byrne et al.   JCI Insight

Development of Highly Efficient Dual‐AAV Split Adenosine Base Editor for In Vivo Gene Therapy

(2020) Yuxi Chen et al.   Small Methods

Endocytosis in cellular uptake of drug delivery vectors: Molecular aspects in drug development

(2020) Zuzanna Kaźmierczak et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Large-Fragment Deletions Induced by Cas9 Cleavage while Not in the BEs System

(2020) Yuning Song et al.   Molecular Therapy-Nucleic Acids

Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing

(2020) Susie Suh et al.   Nature Biomedical Engineering

Current Clinical Applications of In Vivo Gene Therapy with AAVs

(2020) Jerry R. Mendell et al.   MOLECULAR THERAPY

Engineering precision nanoparticles for drug delivery

(2020) Michael J. Mitchell et al.   NATURE REVIEWS DRUG DISCOVERY

Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine

(2020) Lindsey R. Baden et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

(2020) Fernando P. Polack et al.   NEW ENGLAND JOURNAL OF MEDICINE

In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy

(2020) Jennifer B. Kwon 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

Delivering SaCas9 mRNA by lentivirus-like bionanoparticles for transient expression and efficient genome editing

(2019) Baisong Lu 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

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Adopt a moratorium on heritable genome editing

(2019) Eric S. Lander et al.   NATURE

Cell-specific CRISPR–Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins

(2019) Mareike D Hoffmann et al.   NUCLEIC ACIDS RESEARCH

Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

(2019) Ji Liu et al.   ADVANCED MATERIALS

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

(2019) Michael E. Nance et al.   MOLECULAR THERAPY

Delivering Cas9/sgRNA ribonucleoprotein (RNP) by lentiviral capsid-based bionanoparticles for efficient ‘hit-and-run’ genome editing

(2019) Pin Lyu et al.   NUCLEIC ACIDS RESEARCH

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

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

(2019) Andrew V. Anzalone et al.   NATURE

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

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

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

(2019) Akin Akinc et al.   Nature Nanotechnology

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

(2018) Georgia Giannoukos et al.   BMC GENOMICS

Gene therapy clinical trials worldwide to 2017: An update

(2018) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

Clinical use of lentiviral vectors

(2018) Michael C. Milone et al.   LEUKEMIA

Microhomology-mediated end joining: Good, bad and ugly

(2018) Ja-Hwan Seol et al.   MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

(2018) Seuk-Min Ryu et al.   NATURE BIOTECHNOLOGY

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

A modular platform for targeted RNAi therapeutics

(2018) Ranit Kedmi et al.   Nature Nanotechnology

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Viral escape from endosomes and host detection at a glance

(2018) Jacqueline Staring et al.   JOURNAL OF CELL SCIENCE

Gene therapy for neurological disorders: progress and prospects

(2018) Benjamin E. Deverman et al.   NATURE REVIEWS DRUG DISCOVERY

Cas9 immunity creates challenges for CRISPR gene editing therapies

(2018) Julie M. Crudele et al.   Nature Communications

Improved Lentiviral Gene Delivery to Mouse Liver by Hydrodynamic Vector Injection through Tail Vein

(2018) Trine Dalsgaard et al.   Molecular Therapy-Nucleic Acids

Transient retrovirus-based CRISPR/Cas9 all-in-one particles for efficient, targeted gene knockout

(2018) Yvonne Knopp et al.   Molecular Therapy-Nucleic Acids

The Endosomal Escape of Nanoparticles: Toward More Efficient Cellular Delivery

(2018) Samuel A. Smith et al.   BIOCONJUGATE CHEMISTRY

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

(2018) Alireza Edraki et al.   MOLECULAR CELL

Sangamo's landmark genome editing trial gets mixed reception

(2018) Cormac Sheridan   NATURE BIOTECHNOLOGY

High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population

(2018) Dimitrios L. Wagner et al.   NATURE MEDICINE

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

High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing

(2018) Cory D. Sago et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins

(2018) Philippe E. Mangeot et al.   Nature Communications

Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

(2018) Nuphar Veiga et al.   Nature Communications

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

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

In Vivo Hematopoietic Stem Cell Transduction

(2017) Maximilian Richter et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

(2017) Randy J. Chandler et al.   HUMAN GENE THERAPY

CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease

(2017) Su Yang et al.   JOURNAL OF CLINICAL INVESTIGATION

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

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

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

(2017) Nicole M. Gaudelli et al.   NATURE

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Delivery technologies for genome editing

(2017) Hao Yin et al.   NATURE REVIEWS DRUG DISCOVERY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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

Subretinal Injection: A Review on the Novel Route of Therapeutic Delivery for Vitreoretinal Diseases

(2017) Yingqian Peng et al.   OPHTHALMIC RESEARCH

Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics

(2017) James E. Dahlman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2017) Eunji Kim et al.   Nature Communications

Pseudotyped Lentiviral Vectors: One Vector, Many Guises

(2017) Alok V. Joglekar et al.   Human Gene Therapy Methods

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

(2017) Thomas Gaj et al.   Science Advances

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

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

(2016) Jason B. Miller et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Lentivirus pre-packed with Cas9 protein for safer gene editing

(2016) J G Choi et al.   GENE THERAPY

The effect of nanoparticle size on in vivo pharmacokinetics and cellular interaction

(2016) Nazanin Hoshyar et al.   Nanomedicine

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

(2016) Alexis C. Komor et al.   NATURE

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman 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

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

Engineering Delivery Vehicles for Genome Editing

(2016) Christopher E. Nelson et al.   Annual Review of Chemical and Biomolecular Engineering

Mitochondrial diseases

(2016) Gráinne S. Gorman et al.   Nature Reviews Disease Primers

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

Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

(2015) Kevin J. Kauffman et al.   NANO LETTERS

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

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

(2015) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

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

(2015) M. Tabebordbar et al.   SCIENCE

The Blood–Brain Barrier

(2015) Richard Daneman et al.   Cold Spring Harbor Perspectives in Biology

Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes

(2015) Eli J. Fine et al.   Scientific Reports

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

(2015) Eric Zinn et al.   Cell Reports

Highly efficient in vitro and in vivo delivery of functional RNAs using new versatile MS2-chimeric retrovirus-like particles

(2015) Anne Prel et al.   Molecular Therapy-Methods & Clinical Development

Long-Term Episomal Transgene Expression from Mitotically Stable Integration-Deficient Lentiviral Vectors

(2014) Hanna Kymäläinen et al.   HUMAN GENE THERAPY

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

Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector

(2014) Ami M. Kabadi et al.   NUCLEIC ACIDS RESEARCH

Nature's recipe for splitting inteins

(2014) A. S. Aranko et al.   PROTEIN ENGINEERING DESIGN & SELECTION

Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases

(2014) Yujia Cai et al.   eLife

Adeno-associated virus structural biology as a tool in vector development

(2013) Lauren M Drouin et al.   Future Virology

Engraftment of a Galactose Receptor Footprint onto Adeno-associated Viral Capsids Improves Transduction Efficiency

(2013) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

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

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

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

(2012) M. Jinek et al.   SCIENCE

Protein delivery using engineered virus-like particles

(2011) S. J. Kaczmarczyk et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2010) Michael R. Lieber   Annual Review of Biochemistry

Endosomal escape pathways for delivery of biologicals

(2010) Amir K. Varkouhi et al.   JOURNAL OF CONTROLLED RELEASE

Protein transduction from retroviral Gag precursors

(2010) Christine Voelkel et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Integration-deficient Lentiviral Vectors: A Slow Coming of Age

(2009) Klaus Wanisch et al.   MOLECULAR THERAPY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

(2009) Aravind Asokan et al.   NATURE BIOTECHNOLOGY

Mechanism of Eukaryotic Homologous Recombination

(2008) Joseph San Filippo et al.   Annual Review of Biochemistry

Protein Trans-Splicing as a Means for Viral Vector-Mediated In Vivo Gene Therapy

(2008) Juan Li et al.   HUMAN GENE THERAPY

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

(2008) Wuping Li et al.   MOLECULAR THERAPY

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

(2008) Katalin Karikó et al.   MOLECULAR THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE