Genome engineering: a new approach to gene therapy for neuromuscular disorders

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

2017 Clinical trials update: Innovations in hemophilia therapy

(2016) Jan Hartmann et al.   AMERICAN JOURNAL OF HEMATOLOGY

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

(2016) Courtney S. Young et al.   Cell Stem Cell

Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy

(2016) Jacqueline N. Robinson-Hamm et al.   HUMAN GENETICS

CRISPR/dCas9-mediated Transcriptional Inhibition Ameliorates the Epigenetic Dysregulation at D4Z4 and Represses DUX4-fl in FSH Muscular Dystrophy

(2016) Charis L Himeda et al.   MOLECULAR THERAPY

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

(2016) Li Xu et al.   MOLECULAR THERAPY

Genome-editing Technologies for Gene and Cell Therapy

(2016) Morgan L Maeder et al.   MOLECULAR THERAPY

Precise Correction of Disease Mutations in Induced Pluripotent Stem Cells Derived From Patients With Limb Girdle Muscular Dystrophy

(2016) Soeren Turan et al.   MOLECULAR THERAPY

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

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

(2016) Benjamin P. Kleinstiver et al.   NATURE

Applications of CRISPR technologies in research and beyond

(2016) Rodolphe Barrangou et al.   NATURE BIOTECHNOLOGY

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

(2016) Pratiksha I Thakore et al.   NATURE METHODS

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations

(2016) Ignazio Maggio et al.   NUCLEIC ACIDS RESEARCH

Engineering Delivery Vehicles for Genome Editing

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

The emerging role of viral vectors as vehicles for DMD gene editing

(2016) Ignazio Maggio et al.   Genome Medicine

Genome Editing of Monogenic Neuromuscular Diseases

(2016) Chengzu Long et al.   JAMA Neurology

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

(2016) Jean-Paul Iyombe-Engembe et al.   Molecular Therapy-Nucleic Acids

CRISPR genome engineering and viral gene delivery: A case of mutual attraction

(2015) Florian Schmidt et al.   Biotechnology Journal

Therapeutic Approaches for Dominant Muscle Diseases: Highlight on Myotonic Dystrophy

(2015) A. Klein et al.   CURRENT GENE THERAPY

Delivering a disease-modifying treatment for Huntington's disease

(2015) Bruno M.D.C. Godinho et al.   DRUG DISCOVERY TODAY

Progress on Gene Therapy, Cell Therapy, and Pharmacological Strategies Toward the Treatment of Oculopharyngeal Muscular Dystrophy

(2015) Pradeep Harish et al.   HUMAN GENE THERAPY

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

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9

(2015) Yongchang Chen et al.   HUMAN MOLECULAR GENETICS

Safety, tolerability, and pharmacokinetics of SMT C1100, a 2-arylbenzoxazole utrophin modulator, following single- and multiple-dose administration to healthy male adult volunteers

(2015) Jon Tinsley et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Excision of Expanded GAA Repeats Alleviates the Molecular Phenotype of Friedreich's Ataxia

(2015) Yanjie Li et al.   MOLECULAR THERAPY

Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases

(2015) David G Ousterout et al.   MOLECULAR THERAPY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers

(2015) Isaac B Hilton et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

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

Highly specific epigenome editing by CRISPR-Cas9 repressors for silencing of distal regulatory elements

(2015) Pratiksha I Thakore et al.   NATURE METHODS

Spinal muscular atrophy—recent therapeutic advances for an old challenge

(2015) Irene Faravelli et al.   Nature Reviews Neurology

Movement disorders: Friedreich ataxia today—preparing for the final battle

(2015) Giuseppe De Michele et al.   Nature Reviews Neurology

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker 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

Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy Type 1 Induced Pluripotent Stem Cell-Derived Neural Stem Cells

(2015) Guangbin Xia et al.   STEM CELLS

Shortening trinucleotide repeats using highly specific endonucleases: a possible approach to gene therapy?

(2015) Guy-Franck Richard   TRENDS IN GENETICS

Emerging preclinical animal models for FSHD

(2015) Angela Lek et al.   TRENDS IN MOLECULAR MEDICINE

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

(2015) David G. Ousterout et al.   Nature Communications

CRISPR genome engineering and viral gene delivery: A case of mutual attraction

(2015) Florian Schmidt et al.   Biotechnology Journal

Insertion of sequences at the original provirus integration site of mouseROSA26 locus using the CRISPR/Cas9 system

(2015) Rolen M. Quadros et al.   FEBS Open Bio

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

(2015) Hongmei Lisa Li et al.   Stem Cell Reports

Genome Editing in Mouse Spermatogonial Stem Cell Lines Using TALEN and Double-Nicking CRISPR/Cas9

(2015) Takuya Sato et al.   Stem Cell Reports

FHL1 Reduces Dystrophy in Transgenic Mice Overexpressing FSHD Muscular Dystrophy Region Gene 1 (FRG1)

(2015) Sandra J. Feeney et al.   PLoS One

Synthetic Zinc Finger Proteins: The Advent of Targeted Gene Regulation and Genome Modification Technologies

(2014) Charles A. Gersbach et al.   ACCOUNTS OF CHEMICAL RESEARCH

Gene and cell therapy for children — New medicines, new challenges?

(2014) Karen F. Buckland et al.   ADVANCED DRUG DELIVERY REVIEWS

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Distal Myopathies

(2014) Bjarne Udd   Current Neurology and Neuroscience Reports

Efficient gene targeting of theRosa26locus in mouse zygotes using TALE nucleases

(2014) Petr Kasparek et al.   FEBS LETTERS

Efficient gene targeting by homology-directed repair in rat zygotes using TALE nucleases

(2014) Séverine Remy et al.   GENOME RESEARCH

Novel Adeno-Associated Viral Vector Delivering the Utrophin Gene Regulator Jazz Counteracts Dystrophic Pathology in mdx Mice

(2014) Georgios Strimpakos et al.   JOURNAL OF CELLULAR PHYSIOLOGY

High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells

(2014) Yuexin Zhou et al.   NATURE

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese 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

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

(2014) Bin Shen et al.   NATURE METHODS

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Huntington disease: natural history, biomarkers and prospects for therapeutics

(2014) Christopher A. Ross et al.   Nature Reviews Neurology

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

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

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

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

Gene Therapy for Muscular Dystrophy: Moving the Field Forward

(2014) Samiah Al-Zaidy et al.   PEDIATRIC NEUROLOGY

Recombinase-Mediated Reprogramming and Dystrophin Gene Addition in mdx Mouse Induced Pluripotent Stem Cells

(2014) Chunli Zhao et al.   PLoS One

Highly Specific Contractions of a Single CAG/CTG Trinucleotide Repeat by TALEN in Yeast

(2014) Guy-Franck Richard et al.   PLoS One

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Transcription Activator-like Effectors: A Toolkit for Synthetic Biology

(2014) Richard Moore et al.   ACS Synthetic Biology

Adeno-Associated Virus-Mediated Overexpression of LARGE Rescues α-Dystroglycan Function in Dystrophic Mice with Mutations in the Fukutin-Related Protein

(2014) Charles H. Vannoy et al.   Human Gene Therapy Methods

RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice

(2014) Jian Liu et al.   Molecular Therapy-Nucleic Acids

Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9

(2014) David H Stitelman et al.   Molecular Therapy-Methods & Clinical Development

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models

(2014) Catherine Gérard et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated viral vectors do not efficiently target muscle satellite cells

(2014) Andrea LH Arnett et al.   Molecular Therapy-Methods & Clinical Development

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

UtroUp is a novel six zinc finger artificial transcription factor that recognises 18 base pairs of the utrophin promoter and efficiently drives utrophin upregulation

(2013) Annalisa Onori et al.   BMC MOLECULAR BIOLOGY

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

(2013) Haoyi Wang et al.   CELL

CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes

(2013) Luke A. Gilbert et al.   CELL

Adeno-Associated Viral-Mediated LARGE Gene Therapy Rescues the Muscular Dystrophic Phenotype in Mouse Models of Dystroglycanopathy

(2013) Miao Yu et al.   HUMAN GENE THERAPY

Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In

(2013) Linda Popplewell et al.   HUMAN GENE THERAPY

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

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

CpG-depleted adeno-associated virus vectors evade immune detection

(2013) Susan M. Faust et al.   JOURNAL OF CLINICAL INVESTIGATION

Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

(2013) David G Ousterout et al.   MOLECULAR THERAPY

HLA Engineering of Human Pluripotent Stem Cells

(2013) Laura Riolobos et al.   MOLECULAR THERAPY

Morpholino Treatment Improves Muscle Function and Pathology of Pitx1 Transgenic Mice

(2013) Sachchida Nand Pandey et al.   MOLECULAR THERAPY

CD8+ T Cell Recognition of Epitopes Within the Capsid of Adeno-associated Virus 8–based Gene Transfer Vectors Depends on Vectors’ Genome

(2013) Te-Lang Wu et al.   MOLECULAR THERAPY

Treatment of Type 1 Myotonic Dystrophy by Engineering Site-specific RNA Endonucleases that Target (CUG) n Repeats

(2013) Wenjing Zhang et al.   MOLECULAR THERAPY

Autologous Myoblast Transplantation for Oculopharyngeal Muscular Dystrophy: a Phase I/Iia Clinical Study

(2013) Sophie Périé et al.   MOLECULAR THERAPY

Cost of illness for neuromuscular diseases in the United States

(2013) Jane Larkindale et al.   MUSCLE & NERVE

Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library

(2013) Hiroko Koike-Yusa et al.   NATURE BIOTECHNOLOGY

Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins

(2013) Morgan L Maeder et al.   NATURE BIOTECHNOLOGY

RNA-guided gene activation by CRISPR-Cas9–based transcription factors

(2013) Pablo Perez-Pinera et al.   NATURE METHODS

CRISPR RNA–guided activation of endogenous human genes

(2013) Morgan L Maeder et al.   NATURE METHODS

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

(2013) Richard S. Finkel et al.   PLoS One

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Genetic Screens in Human Cells Using the CRISPR-Cas9 System

(2013) Tim Wang et al.   SCIENCE

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation

(2013) Basma F Benabdallah et al.   Molecular Therapy-Nucleic Acids

A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins

(2013) Pierre Chapdelaine et al.   Molecular Therapy-Nucleic Acids

Targeted Myostatin Gene Editing in Multiple Mammalian Species Directed by a Single Pair of TALE Nucleases

(2013) Li Xu et al.   Molecular Therapy-Nucleic Acids

Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice

(2012) Radbod Darabi et al.   Cell Stem Cell

Gene Replacement Therapies for Duchenne Muscular Dystrophy Using Adeno-Associated Viral Vectors

(2012) Jane T. Seto et al.   CURRENT GENE THERAPY

RNAi-based Gene Therapy for Dominant Limb Girdle Muscular Dystrophies

(2012) Jian Liu et al.   CURRENT GENE THERAPY

Gene therapy for Duchenne muscular dystrophy

(2012) Ingrid E.C. Verhaart et al.   CURRENT OPINION IN NEUROLOGY

Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo

(2012) N A McNeer et al.   GENE THERAPY

Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy

(2012) Helen Foster et al.   HUMAN GENE THERAPY

Europe gives gene therapy the green light

(2012) Karl Gruber   LANCET

The myotonic dystrophies: molecular, clinical, and therapeutic challenges

(2012) Bjarne Udd et al.   LANCET NEUROLOGY

RNA Interference Inhibits DUX4-induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy

(2012) Lindsay M Wallace et al.   MOLECULAR THERAPY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Chimeric TALE recombinases with programmable DNA sequence specificity

(2012) Andrew C. Mercer et al.   NUCLEIC ACIDS RESEARCH

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells

(2012) Maarten Holkers et al.   NUCLEIC ACIDS RESEARCH

Evaluation of OPEN Zinc Finger Nucleases for Direct Gene Targeting of the ROSA26 Locus in Mouse Embryos

(2012) Mario Hermann et al.   PLoS One

Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice

(2012) M. Garriga-Canut 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

Genetic Correction of Human Induced Pluripotent Stem Cells from Patients with Spinal Muscular Atrophy

(2012) S. Corti et al.   Science Translational Medicine

Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy

(2012) F. S. Tedesco et al.   Science Translational Medicine

Comprehensive interrogation of natural TALE DNA-binding modules and transcriptional repressor domains

(2012) Le Cong et al.   Nature Communications

Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy

(2011) George Silva et al.   CURRENT GENE THERAPY

Dose Response in Rodents and Nonhuman Primates After Hydrodynamic Limb Vein Delivery of Naked Plasmid DNA

(2011) Christine I. Wooddell et al.   HUMAN GENE THERAPY

Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

(2011) Yadong Zhang et al.   HUMAN GENE THERAPY

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

(2011) Paul N. Porensky et al.   HUMAN MOLECULAR GENETICS

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

AAV6-mediated Systemic shRNA Delivery Reverses Disease in a Mouse Model of Facioscapulohumeral Muscular Dystrophy

(2011) Sergia Bortolanza et al.   MOLECULAR THERAPY

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

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

(2011) Elitza Deltcheva et al.   NATURE

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

(2011) Hojun Li et al.   NATURE

Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription

(2011) Feng Zhang et al.   NATURE BIOTECHNOLOGY

Evolution and classification of the CRISPR–Cas systems

(2011) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

The collagen VI-related myopathies: muscle meets its matrix

(2011) Carsten G. Bönnemann   Nature Reviews Neurology

Distal myopathies – New genetic entities expand diagnostic challenge

(2011) Bjarne Udd   NEUROMUSCULAR DISORDERS

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficient gene targeting mediated by a lentiviral vector-associated meganuclease

(2011) Araksya Izmiryan et al.   NUCLEIC ACIDS RESEARCH

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

(2011) Tomas Cermak et al.   NUCLEIC ACIDS RESEARCH

Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases

(2011) Pablo Perez-Pinera et al.   NUCLEIC ACIDS RESEARCH

Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse

(2011) Jonathon M. Tinsley et al.   PLoS One

TAL Effectors: Customizable Proteins for DNA Targeting

(2011) A. J. Bogdanove et al.   SCIENCE

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

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

(2011) Barry L. Stoddard   STRUCTURE

DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivo

(2010) Lindsay M. Wallace et al.   ANNALS OF NEUROLOGY

Meganucleases can restore the reading frame of a mutated dystrophin

(2010) P Chapdelaine et al.   GENE THERAPY

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   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

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

(2010) William Lostal et al.   HUMAN MOLECULAR GENETICS

Lentiviral Vector Gene Therapy: Effective and Safe?

(2010) Derek A Persons   MOLECULAR THERAPY

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

(2010) Qi-Long Lu et al.   MOLECULAR THERAPY

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Genome editing with engineered zinc finger nucleases

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

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion

(2010) Cem Şöllü et al.   NUCLEIC ACIDS RESEARCH

Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice

(2010) A. R. Amenta et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Regulation of selected genome loci using de novo-engineered transcription activator-like effector (TALE)-type transcription factors

(2010) R. Morbitzer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Unifying Genetic Model for Facioscapulohumeral Muscular Dystrophy

(2010) R. J. L. F. Lemmers et al.   SCIENCE

A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy

(2010) M. Krahn et al.   Science Translational Medicine

Myoblast transplantation: A possible surgical treatment for a severe pediatric disease

(2010) Beniamino Palmieri et al.   SURGERY TODAY

Myofibrillar Myopathies: A Clinical and Myopathological Guide

(2009) Rolf Schröder et al.   BRAIN PATHOLOGY

Targeted chromosomal deletions in human cells using zinc finger nucleases

(2009) H. J. Lee et al.   GENOME RESEARCH

Functional Efficacy of Dystrophin Expression from Plasmids Delivered to mdx Mice by Hydrodynamic Limb Vein Injection

(2009) Guofeng Zhang et al.   HUMAN GENE THERAPY

The artificial gene Jazz, a transcriptional regulator of utrophin, corrects the dystrophic pathology in mdx mice

(2009) Maria Grazia Di Certo et al.   HUMAN MOLECULAR GENETICS

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Adeno-associated virus serotype 2 induces cell-mediated immune responses directed against multiple epitopes of the capsid protein VP1

(2009) D. Madsen et al.   JOURNAL OF GENERAL VIROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression

(2009) En Kimura 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

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection

(2009) L. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Synthesis of programmable integrases

(2009) R. M. Gordley et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Zinc-finger directed double-strand breaks within CAG repeat tracts promote repeat instability in human cells

(2009) D. Mittelman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

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

(2008) Wuping Li et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Small CRISPR RNAs Guide Antiviral Defense in Prokaryotes

(2008) S. J. J. Brouns et al.   SCIENCE

Genetic conversion of an SMN2 gene to SMN1: A novel approach to the treatment of spinal muscular atrophy

(2007) Darlise DiMatteo et al.   EXPERIMENTAL CELL RESEARCH