CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

Exon-skipping advances for Duchenne muscular dystrophy

(2018) Lucía Echevarría et al.   HUMAN MOLECULAR GENETICS

Eteplirsen treatment for Duchenne muscular dystrophy

(2018) Jay S. Charleston et al.   NEUROLOGY

CRISPR/Cas9 cleavages in budding yeast reveal templated insertions and strand-specific insertion/deletion profiles

(2018) Brenda R. Lemos et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

(2018) Chengzu Long et al.   Science Advances

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

(2018) Leonela Amoasii et al.   SCIENCE

Unexpected mutations after CRISPR–Cas9 editing in vivo

(2017) Kellie A Schaefer et al.   NATURE METHODS

Espectro mutacional de la distrofia muscular de Duchenne en España: estudio de 284 casos

(2017) I. Vieitez et al.   NEUROLOGIA

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy

(2016) David G. Allen et al.   PHYSIOLOGICAL REVIEWS

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

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

DMD mutation spectrum analysis in 613 Chinese patients with dystrophinopathy

(2015) Ruolan Guo et al.   JOURNAL OF HUMAN GENETICS

117. AAV-TRISPR – A Novel Versatile AAV Vector Kit for Combinatorial CRISPR and RNAi Expression

(2015) Florian Schmidt 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

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation

(2014) M. Jinek et al.   SCIENCE

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

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

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Comparative Cardiac Gene Delivery of Adeno-Associated Virus Serotypes 1-9 reveals that AAV6 Mediates the Most Efficient Transduction in Mouse Heart

(2010) Carmela Zincarelli et al.   CTS-Clinical and Translational Science

Mutation spectrum of the dystrophin gene in 442 Duchenne/Becker muscular dystrophy cases from one Japanese referral center

(2010) Yasuhiro Takeshima et al.   JOURNAL OF HUMAN GENETICS

Partial Rescue of Growth Failure in Growth Hormone (GH)-Deficient Mice by a Single Injection of a Double-Stranded Adeno-Associated Viral Vector Expressing the GH Gene Driven by a Muscle-Specific Regulatory Cassette

(2009) Marco Martari et al.   HUMAN GENE THERAPY

Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort

(2009) Kevin M. Flanigan et al.   HUMAN MUTATION

Recent developments in adeno‐associated virus vector technology

(2008) Hildegard Büning et al.   JOURNAL OF GENE MEDICINE

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY