Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells

sPIF promotes myoblast differentiation and utrophin expression while inhibiting fibrosis in Duchenne muscular dystrophy via the H19/miR-675/let-7 and miR-21 pathways

(2019) Daria Morgoulis et al.   Cell Death & Disease

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantation

(2019) Alberto Daniel-Moreno et al.   BONE MARROW TRANSPLANTATION

In Situ Modification of Tissue Stem and Progenitor Cell Genomes

(2019) Jill M. Goldstein et al.   Cell Reports

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

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

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

(2019) Yafeng Song et al.   NATURE MEDICINE

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

Exon-skipping advances for Duchenne muscular dystrophy

(2018) Lucía Echevarría et al.   HUMAN MOLECULAR GENETICS

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering

(2018) Sara Martina Maffioletti et al.   Cell Reports

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Induced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and Therapy

(2018) Lubos Danisovic et al.   Cells

Protein-Anchoring Therapy of Biglycan for Mdx Mouse Model of Duchenne Muscular Dystrophy

(2017) Mikako Ito et al.   HUMAN GENE THERAPY

Progress toward Gene Therapy for Duchenne Muscular Dystrophy

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

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

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

CRISPR/Cas9 Genome-Editing System in Human Stem Cells: Current Status and Future Prospects

(2017) Zhao Zhang et al.   Molecular Therapy-Nucleic Acids

Functional improvement of dystrophic muscle by repression of utrophin: let-7c interaction

(2017) Manoj K. Mishra et al.   PLoS One

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

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

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

Prevention of Muscle Wasting by CRISPR/Cas9-mediated Disruption of Myostatin In Vivo

(2016) Yuda Wei et al.   MOLECULAR THERAPY

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

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

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

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy

(2014) Jean K. Mah et al.   NEUROMUSCULAR DISORDERS

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

(2014) C. Long et al.   SCIENCE

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

(2014) Thomas J Cradick et al.   Molecular Therapy-Nucleic Acids

Gene therapy for muscular dystrophy: Lessons learned and path forward

(2012) Jerry R. Mendell et al.   NEUROSCIENCE LETTERS

From Skin Biopsy to Neurons Through a Pluripotent Intermediate Under Good Manufacturing Practice Protocols

(2012) Saravanan Karumbayaram et al.   Stem Cells Translational Medicine

Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

(2011) Sebahattin Cirak et al.   MOLECULAR THERAPY

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

Translational Regulation of Utrophin by miRNAs

(2011) Utpal Basu et al.   PLoS One

Drug Discovery for Duchenne Muscular Dystrophy via Utrophin Promoter Activation Screening

(2011) Catherine Moorwood et al.   PLoS One

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

Nanog Is the Gateway to the Pluripotent Ground State

(2009) Jose Silva et al.   CELL

Long-Term Benefit of Adeno-Associated Virus/Antisense-Mediated Exon Skipping in Dystrophic Mice

(2008) Michela Alessandra Denti et al.   HUMAN GENE THERAPY

Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice

(2008) Guy L Odom et al.   MOLECULAR THERAPY