Biological and genetic therapies for the treatment of Duchenne muscular dystrophy

CRISPR Therapeutics for Duchenne Muscular Dystrophy

(2022) Esra Erkut et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

(2022) Craig M McDonald et al.   LANCET

Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy

(2022) Kenji Rowel Q. Lim et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Anomalous collapses of Nares Strait ice arches leads to enhanced export of Arctic sea ice

(2021) G. W. K. Moore et al.   Nature Communications

Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy

(2021) Vratko Himič et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Duchenne muscular dystrophy

(2021) Dongsheng Duan et al.   Nature Reviews Disease Primers

Casimersen: First Approval

(2021) Matt Shirley   DRUGS

CRISPR technologies for the treatment of Duchenne muscular dystrophy

(2021) Eunyoung Choi et al.   MOLECULAR THERAPY

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

(2021) Kiran Musunuru et al.   NATURE

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

Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial

(2021) Kathryn R. Wagner et al.   MUSCLE & NERVE

The use of umbilical cord‐derived mesenchymal stem cells in patients with muscular dystrophies: Results from compassionate use in real‐life settings

(2021) Beata Świątkowska‐Flis et al.   Stem Cells Translational Medicine

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

(2021) Matthew Behr et al.   Acta Pharmaceutica Sinica B

Fourth Boy Dies in Clinical Trial of Astellas' AT132

(2021) Alex Philippidis   HUMAN GENE THERAPY

Life Expectancy in Duchenne Muscular Dystrophy

(2021) Jonathan Broomfield et al.   NEUROLOGY

Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)

(2020) M.W. Konstan et al.   Journal of Cystic Fibrosis

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

(2020) Diane E. Frank et al.   NEUROLOGY

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

(2020) Eugenio Mercuri et al.   Journal of Comparative Effectiveness Research

The Genetic Landscape of Dystrophin Mutations in Italy: A Nationwide Study

(2020) Marcella Neri et al.   Frontiers in Genetics

Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing

(2020) Yi-Li Min et al.   MOLECULAR THERAPY

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

(2020) Paula R. Clemens et al.   JAMA Neurology

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

(2020) Giuseppe Ronzitti et al.   Frontiers in Immunology

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

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

Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis

(2020) Salvatore Crisafulli et al.   Orphanet Journal of Rare Diseases

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

(2020) Tuo Wei et al.   Nature Communications

Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing

(2020) Muhammad Naeem et al.   Cells

After Third Death, Audentes' AT132 Remains on Clinical Hold

(2020) Alex Philippidis   HUMAN GENE THERAPY

Duchenne Dilated Cardiomyopathy: Cardiac Management from Prevention to Advanced Cardiovascular Therapies

(2020) Rachele Adorisio et al.   Journal of Clinical Medicine

A simple method using CRISPR-Cas9 to knock-out genes in murine cancerous cell lines

(2020) Airi Ishibashi et al.   Scientific Reports

rAAVrh74.MCK.GALGT2 Protects Against Loss of Hemodynamic Function in the Aging mdx Mouse Heart

(2019) Rui Xu et al.   MOLECULAR THERAPY

Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial

(2019) Michael Taylor et al.   NEUROLOGY

In vivo RNA editing of point mutations via RNA-guided adenosine deaminases

(2019) Dhruva Katrekar et al.   NATURE METHODS

Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the DMD gene

(2019) Yusuke Echigoya et al.   MOLECULAR THERAPY

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

(2019) Andrew V. Anzalone et al.   NATURE

Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models

(2019) Yafeng Song et al.   NATURE MEDICINE

Stem cell-based therapies for Duchenne muscular dystrophy

(2019) Congshan Sun et al.   EXPERIMENTAL NEUROLOGY

rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice

(2019) Deborah A. Zygmunt et al.   Molecular Therapy-Methods & Clinical Development

CRISPR RNAs trigger innate immune responses in human cells

(2018) Sojung Kim et al.   GENOME RESEARCH

Micro-Dystrophin Gene Therapy Goes Systemic in Duchenne Muscular Dystrophy Patients

(2018) Dongsheng Duan   HUMAN GENE THERAPY

Exon-skipping advances for Duchenne muscular dystrophy

(2018) Lucía Echevarría et al.   HUMAN MOLECULAR GENETICS

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons

(2018) Maciej Dabrowski et al.   MOLECULAR MEDICINE

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

The CRISPR tool kit for genome editing and beyond

(2018) Mazhar Adli   Nature Communications

Antisense Phosphorodiamidate Morpholino Oligomers as Novel Antiviral Compounds

(2018) Yuchen Nan et al.   Frontiers in Microbiology

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

A mini review and implementation model for using ataluren to treat nonsense mutation Duchenne muscular dystrophy

(2018) Erik Landfeldt et al.   ACTA PAEDIATRICA

CRISPR Correction of Duchenne Muscular Dystrophy

(2018) Yi-Li Min et al.   Annual Review of Medicine

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

(2018) Juanjuan Yuan et al.   MOLECULAR CELL

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

PhaseOut DMD: a Phase 2, proof of concept, clinical study of utrophin modulation with ezutromid

(2017) F. Muntoni et al.   NEUROMUSCULAR DISORDERS

The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

(2017) S. Ryder et al.   Orphanet Journal of Rare Diseases

In situ click chemistry generation of cyclooxygenase-2 inhibitors

(2017) Atul Bhardwaj et al.   Nature Communications

Eteplirsen in the treatment of Duchenne muscular dystrophy

(2017) Kenji Rowel Lim et al.   Drug Design Development and Therapy

CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy

(2017) Pei Zhu et al.   Molecular Therapy-Nucleic Acids

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Deletion of exons 3−9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy

(2016) Akinori Nakamura et al.   JOURNAL OF HUMAN GENETICS

Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9

(2016) John G Doench et al.   NATURE BIOTECHNOLOGY

Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy

(2016) David Gloss et al.   NEUROLOGY

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

Systematic quantification of HDR and NHEJ reveals effects of locus, nuclease, and cell type on genome-editing

(2016) Yuichiro Miyaoka et al.   Scientific Reports

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

(2015) Xiao-Hui Zhang et al.   Molecular Therapy-Nucleic Acids

Duchenne muscular dystrophy: current cell therapies

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

EMA reconsiders 'read-through' drug against Duchenne muscular dystrophy following appeal

(2014) Asher Mullard   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

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

(2014) C. Long et al.   SCIENCE

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

(2013) F. Mingozzi et al.   BLOOD

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

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

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

(2012) Y. Aoki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Dystrophin: More than just the sum of its parts

(2010) Elisabeth Le Rumeur et al.   BIOCHIMICA ET BIOPHYSICA ACTA-PROTEINS AND PROTEOMICS

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

P3.51 Results of a Phase 2b, dose-ranging study of ataluren (PTC124®) in nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD)

(2010) R. Finkel et al.   NEUROMUSCULAR DISORDERS

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy

(2010) Vinod Malik et al.   Therapeutic Advances in Neurological Disorders

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

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

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