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

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

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

In Situ Modification of Tissue Stem and Progenitor Cell Genomes

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

Update in Duchenne and Becker muscular dystrophy

(2019) Megan A. Waldrop et al.   CURRENT OPINION IN NEUROLOGY

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

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

Muscle satellite cells and impaired late stage regeneration in different murine models for muscular dystrophies

(2019) Antonio F. Ribeiro et al.   Scientific Reports

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

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

(2018) Leonela Amoasii et al.   SCIENCE

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

(2017) Christopher E. Nelson et al.   Nature Reviews Neurology

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

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

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

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

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

(2016) Li Xu et al.   MOLECULAR THERAPY

Engineering Delivery Vehicles for Genome Editing

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

Engineering AAV receptor footprints for gene therapy

(2016) Victoria J Madigan et al.   Current Opinion in Virology

Comparative Study of Injury Models for Studying Muscle Regeneration in Mice

(2016) David Hardy et al.   PLoS One

Intrinsic and extrinsic mechanisms regulating satellite cell function

(2015) N. A. Dumont et al.   DEVELOPMENT

Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division

(2015) Nicolas A Dumont et al.   NATURE MEDICINE

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

Muscle stem cells contribute to myofibres in sedentary adult mice

(2015) Alexandra C. Keefe et al.   Nature Communications

Notch signaling deficiency underlies age-dependent depletion of satellite cells in muscular dystrophy

(2014) C. Jiang et al.   Disease Models & Mechanisms

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

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

Embryonic founders of adult muscle stem cells are primed by the determination gene Mrf4

(2013) Ramkumar Sambasivan et al.   DEVELOPMENTAL BIOLOGY

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

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

Revertant Fibers in the mdx Murine Model of Duchenne Muscular Dystrophy: An Age- and Muscle-Related Reappraisal

(2013) Sarah R. Pigozzo et al.   PLoS One

A Contemporary Atlas of the Mouse Diaphragm

(2012) Pascal Stuelsatz et al.   JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY

Donor Satellite Cell Engraftment Is SignificantlyAugmented When the Host Niche Is Preserved and Endogenous Satellite Cells Are Incapacitated

(2012) Luisa Boldrin et al.   STEM CELLS

Short Telomeres and Stem Cell Exhaustion Model Duchenne Muscular Dystrophy in mdx/mTR Mice

(2010) Alessandra Sacco et al.   CELL

A robust and high-throughput Cre reporting and characterization system for the whole mouse brain

(2009) Linda Madisen et al.   NATURE NEUROSCIENCE

Transduction Efficiencies of Novel AAV Vectors in Mouse Airway Epithelium In Vivo and Human Ciliated Airway Epithelium In Vitro

(2008) Maria P Limberis et al.   MOLECULAR THERAPY