Common therapeutic advances for Duchenne muscular dystrophy (DMD)

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

(2019) Andrew V. Anzalone et al.   NATURE

Utrophin up-regulation by artificial transcription factors induces muscle rescue and impacts the neuromuscular junction in mdx mice

(2018) Cinzia Pisani et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

CRISPR-Based Chromatin Remodeling of the Endogenous Oct4 or Sox2 Locus Enables Reprogramming to Pluripotency

(2018) Peng Liu et al.   Cell Stem Cell

Cardiosphere-derived cells show strong immunomodulatory activity and improve muscle physiology when systemically delivered in a DMD mouse model

(2018) J.L. Moseley et al.   CYTOTHERAPY

Exon-skipping advances for Duchenne muscular dystrophy

(2018) Lucía Echevarría et al.   HUMAN MOLECULAR GENETICS

Duchenne muscular dystrophy: an updated review of common available therapies

(2018) Arash Salmaninejad et al.   INTERNATIONAL JOURNAL OF NEUROSCIENCE

A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping

(2018) Takenori Shimo et al.   JOURNAL OF HUMAN GENETICS

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

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

Base editing with a Cpf1–cytidine deaminase fusion

(2018) Xiaosa Li et al.   NATURE BIOTECHNOLOGY

Golodirsen induces exon skipping leading to sarcolemmal dystrophin expression in patients with genetic mutations amenable to exon 53 skipping

(2018) F. Muntoni et al.   NEUROMUSCULAR DISORDERS

Genetic Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase

(2018) Juanjuan Yuan et al.   MOLECULAR CELL

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

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

The CRISPR Growth Spurt: from Bench to Clinic on Versatile Small RNAs

(2017) Hadi Bayat et al.   JOURNAL OF MICROBIOLOGY AND BIOTECHNOLOGY

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

(2017) Craig M McDonald et al.   LANCET

Progress toward Gene Therapy for Duchenne Muscular Dystrophy

(2017) Joel R. Chamberlain et al.   MOLECULAR THERAPY

Stunning pharmacological properties of DS-5141b, an antisense oligonucleotide consisting of 2'-O,4'-C-ethylene-bridged nucleic acids and 2'-O-methyl RNA, on dystrophin mRNA exon skipping

(2017) K. Takaishi et al.   NEUROMUSCULAR DISORDERS

SGT-001 Micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2017) J. Schneider et al.   NEUROMUSCULAR DISORDERS

WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration

(2017) M. Wood et al.   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

Clinical trials of exon skipping in Duchenne muscular dystrophy

(2017) Jerry R Mendell et al.   Expert Opinion on Orphan Drugs

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System

(2017) Annalisa Lattanzi et al.   Molecular Therapy-Nucleic Acids

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

(2017) Yu Zhang et al.   Science Advances

Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders

(2016) Daria Wojtal et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Phosphorylation of eIF2α Is a Translational Control Mechanism Regulating Muscle Stem Cell Quiescence and Self-Renewal

(2016) Victoria Zismanov et al.   Cell Stem Cell

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

Next stop for the CRISPR revolution: RNA-guided epigenetic regulators

(2016) Suhani Vora et al.   FEBS Journal

Increasing taurine intake and taurine synthesis improves skeletal muscle function in the mdx mouse model for Duchenne muscular dystrophy

(2016) Jessica R. Terrill et al.   JOURNAL OF PHYSIOLOGY-LONDON

379. MicroRNA-29 and Micro-Dystrophin Combinatorial Therapy Suppresses Fibrosis and Restores Function to mdx/utrn+/− Mice

(2016) Kristin Heller 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

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

Treatment with l-citrulline and metformin in Duchenne muscular dystrophy: study protocol for a single-centre, randomised, placebo-controlled trial

(2016) Patricia Hafner et al.   Trials

Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy

(2016) Jinhong Meng et al.   Scientific Reports

Concise Review: Epigenetic Regulation of Myogenesis in Health and Disease

(2016) Marie-Claude Sincennes et al.   Stem Cells Translational Medicine

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

Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

(2016) Valeria Ricotti et al.   PLoS One

The Pathogenesis and Therapy of Muscular Dystrophies

(2015) Simon Guiraud et al.   Annual Review of Genomics and Human Genetics

Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy

(2015) G. Cossu et al.   EMBO Molecular Medicine

Advances in genetic therapeutic strategies for Duchenne muscular dystrophy

(2015) Simon Guiraud et al.   EXPERIMENTAL PHYSIOLOGY

Combinatorial therapeutic activation with heparin and AICAR stimulates additive effects on utrophin A expression in dystrophic muscles

(2015) Christine Péladeau et al.   HUMAN MOLECULAR GENETICS

Second-generation compound for the modulation of utrophin in the therapy of DMD

(2015) Simon Guiraud et al.   HUMAN MOLECULAR GENETICS

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

Taurine deficiency, synthesis and transport in the mdx mouse model for Duchenne Muscular Dystrophy

(2015) Jessica R. Terrill et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

(2015) Jerry R Mendell 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

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

(2015) Aurélie Goyenvalle et al.   NATURE MEDICINE

Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells

(2015) Sara M Maffioletti et al.   Nature Protocols

Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01: A phase 1, dose escalation, first-in-human study

(2015) H. Komaki et al.   NEUROMUSCULAR DISORDERS

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

Human cancer immunotherapy with antibodies to the PD-1 and PD-L1 pathway

(2015) Kim C. Ohaegbulam 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

Chromatin signaling in muscle stem cells: interpreting the regenerative microenvironment

(2015) Arianna Brancaccio et al.   Frontiers in Aging Neuroscience

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

Duchenne muscular dystrophy: current cell therapies

(2015) Dorota Sienkiewicz et al.   Therapeutic Advances in Neurological Disorders

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

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

TAL effectors: tools for DNA Targeting

(2014) R. Jankele et al.   Briefings in Functional Genomics

A Population-Based Study of Dystrophin Mutations in Canada

(2014) Jean K. Mah et al.   CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES

Histological and biochemical outcomes of cardiac pathology inmdxmice with dietary quercetin enrichment

(2014) Christopher Ballmann et al.   EXPERIMENTAL PHYSIOLOGY

Utrophin A is essential in mediating the functional adaptations of mdx mouse muscle following chronic AMPK activation

(2014) H. Al-Rewashdy et al.   HUMAN MOLECULAR GENETICS

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

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

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

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

(2014) C. Long et al.   SCIENCE

Exercise restores decreased physical activity levels and increases markers of autophagy and oxidative capacity in myostatin/activin-blocked mdx mice

(2013) Juha J. Hulmi et al.   AMERICAN JOURNAL OF PHYSIOLOGY-ENDOCRINOLOGY AND METABOLISM

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Robust ZFN-mediated genome editing in adult hemophilic mice

(2013) X. M. Anguela et al.   BLOOD

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

(2013) Lei S. Qi et al.   CELL

Arginine butyrate: a therapeutic candidate for Duchenne muscular dystrophy

(2013) Sara Vianello et al.   FASEB JOURNAL

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

(2013) Linda Popplewell et al.   HUMAN GENE THERAPY

The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia

(2013) Catherine L. Bladen et al.   HUMAN MUTATION

Corticosteroids Can Reduce the Severity of Scoliosis in Duchenne Muscular Dystrophy

(2013) Anne M Connolly et al.   JOURNAL OF BONE AND JOINT SURGERY-AMERICAN VOLUME

Evaluation of suppressive and pro-resolving effects of EPA and DHA in human primary monocytes and T-helper cells

(2013) Anke Jaudszus et al.   JOURNAL OF LIPID RESEARCH

Oral Corticosteroids and Onset of Cardiomyopathy in Duchenne Muscular Dystrophy

(2013) Brent J. Barber et al.   JOURNAL OF PEDIATRICS

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and othe

(2013) Erik K. Henricson et al.   MUSCLE & NERVE

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

(2013) Rebecca J. Fairclough et al.   NATURE REVIEWS GENETICS

megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering

(2013) Sandrine Boissel et al.   NUCLEIC ACIDS RESEARCH

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

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

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

Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

(2013) Yoshitsugu Aoki et al.   Biomed Research International

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

Review of Phase II and Phase III clinical trials for Duchenne muscular dystrophy

(2013) Michele A Scully et al.   Expert Opinion on Orphan Drugs

Evidence-based path to newborn screening for duchenne muscular dystrophy

(2012) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Carm1 Regulates Pax7 Transcriptional Activity through MLL1/2 Recruitment during Asymmetric Satellite Stem Cell Divisions

(2012) Yoh-ichi Kawabe et al.   Cell Stem Cell

Administration of a Soluble Activin Type IIB Receptor Promotes the Transplantation of Human Myoblasts in Dystrophic Mice

(2012) Raouia Fakhfakh et al.   CELL TRANSPLANTATION

Resveratrol decreases inflammation and increases utrophin gene expression in the mdx mouse model of duchenne muscular dystrophy

(2012) Bradley S. Gordon et al.   CLINICAL NUTRITION

‘Hearts and bones’: the ups and downs of ‘plasticity’ in stem cell biology

(2012) Paola Bonfanti et al.   EMBO Molecular Medicine

Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy

(2012) Helen Foster et al.   HUMAN GENE THERAPY

Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer in mdx Mice

(2012) Stefanie Schinkel et al.   HUMAN GENE THERAPY

Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy

(2012) Refik Kayali et al.   HUMAN MOLECULAR GENETICS

Notch signaling regulates PD-1 expression during CD8+ T-cell activation

(2012) Mélissa Mathieu et al.   IMMUNOLOGY AND CELL BIOLOGY

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

(2012) Adeline Vulin et al.   MOLECULAR THERAPY

Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy

(2012) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

TALENs: a widely applicable technology for targeted genome editing

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

Pharmaceutical therapies to recode nonsense mutations in inherited diseases

(2012) Hui-Ling Rose Lee et al.   PHARMACOLOGY & THERAPEUTICS

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

Experimental Gentamicin Nephrotoxicity and Agents that Modify it: A Mini-Review of Recent Research

(2011) Badreldin H. Ali et al.   BASIC & CLINICAL PHARMACOLOGY & TOXICOLOGY

Effective Drug Delivery System for Duchenne Muscular Dystrophy Using Hybrid Liposomes Including Gentamicin along with Reduced Toxicity

(2011) Mamiko Yukihara et al.   BIOLOGICAL & PHARMACEUTICAL BULLETIN

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells

(2011) Kosuke Yusa et al.   NATURE

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

(2011) Hojun Li et al.   NATURE

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

Readthrough of Premature Termination Codons in the Adenomatous Polyposis Coli Gene Restores Its Biological Activity in Human Cancer Cells

(2011) Célia Floquet et al.   PLoS One

Drug Discovery for Duchenne Muscular Dystrophy via Utrophin Promoter Activation Screening

(2011) Catherine Moorwood et al.   PLoS One

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

Repairing faulty genes by aminoglycosides: Development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations

(2010) Igor Nudelman et al.   BIOORGANIC & MEDICINAL CHEMISTRY

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

Administration of a soluble activin type IIB receptor promotes skeletal muscle growth independent of fiber type

(2010) Samuel M. Cadena et al.   JOURNAL OF APPLIED PHYSIOLOGY

Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne/ Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)

(2010) Richard S. Finkel   JOURNAL OF CHILD NEUROLOGY

Use of Corticosteroids in a Population-Based Cohort of Boys With Duchenne and Becker Muscular Dystrophy

(2010) Dennis J. Matthews et al.   JOURNAL OF CHILD NEUROLOGY

CT-GalNAc transferase overexpression in adult mice is associated with extrasynaptic utrophin in skeletal muscle fibres

(2010) Margaret Durko et al.   JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Identification and characterization of a non-satellite cell muscle resident progenitor during postnatal development

(2010) Kathryn J. Mitchell et al.   NATURE CELL BIOLOGY

Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures

(2010) Yannick Doyon et al.   NATURE METHODS

Genome editing with engineered zinc finger nucleases

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

Evaluation of potential synergistic action of a combined treatment with alpha-methyl-prednisolone and taurine on the mdx mouse model of Duchenne muscular dystrophy

(2010) A. Cozzoli et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion

(2010) Cem Şöllü et al.   NUCLEIC ACIDS RESEARCH

TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain

(2010) Ting Li 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

Reprogramming of Human Umbilical Cord Stromal Mesenchymal Stem Cells for Myogenic Differentiation and Muscle Repair

(2010) Çetin Kocaefe et al.   Stem Cell Reviews and Reports

Interaction of Aminoglycosides with Human Mitochondrial 12S rRNA Carrying the Deafness-Associated Mutation

(2009) Y. Qian et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Mesenchymal stem cells as anti-inflammatories: Implications for treatment of Duchenne muscular dystrophy

(2009) Thomas E. Ichim et al.   CELLULAR IMMUNOLOGY

Pharmacological activation of PPAR /  stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx mice

(2009) P. Miura 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

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

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Exon-skipping therapy for Duchenne muscular dystrophy

(2009) Akinori Nakamura et al.   NEUROPATHOLOGY

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

(2009) J. Boch et al.   SCIENCE

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy

(2008) Kathryn R. Wagner et al.   ANNALS OF NEUROLOGY

Targeting Artificial Transcription Factors to the Utrophin A Promoter

(2008) Yifan Lu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Expanding or Restricting the Target Site Repertoire of Zinc-finger Nucleases: The Inter-domain Linker as a Major Determinant of Target Site Selectivity

(2008) Eva-Maria Händel et al.   MOLECULAR THERAPY

Functional skeletal muscle regeneration from differentiating embryonic stem cells

(2008) Radbod Darabi et al.   NATURE MEDICINE

Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors

(2008) A. M. Haidet et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice

(2007) Masashi Nakatani et al.   FASEB JOURNAL

Drug-induced readthrough of premature stop codons leads to the stabilization of laminin α2 chain mRNA in CMD myotubes

(2007) Valérie Allamand et al.   JOURNAL OF GENE MEDICINE