A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9

CRISPR/Cas9-mediated mutation ofPHEXin rabbit recapitulates human X-linked hypophosphatemia (XLH)

(2016) Tingting Sui et al.   HUMAN MOLECULAR GENETICS

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

(2016) Li Xu et al.   MOLECULAR THERAPY

Empower multiplex cell and tissue-specific CRISPR-mediated gene manipulation with self-cleaving ribozymes and tRNA

(2016) Li Xu et al.   NUCLEIC ACIDS RESEARCH

CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts

(2016) Lin Yuan et al.   Scientific Reports

Single-step generation of rabbits carrying a targeted allele of the tyrosinase gene using CRISPR/Cas9

(2015) Arata HONDA et al.   EXPERIMENTAL ANIMALS

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

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

Genetic disruption of Ano5 in mice does not recapitulate human ANO5-deficient muscular dystrophy

(2015) Jing Xu et al.   Skeletal Muscle

Generation of gene-modified mice via Cas9/RNA-mediated gene targeting

(2013) Bin Shen et al.   CELL RESEARCH

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

(2013) Nikolai Klymiuk et al.   HUMAN MOLECULAR GENETICS

Novel mutation in exon 56 of the dystrophin gene in a child with Duchenne muscular dystrophy

(2013) JIAN-FANG ZHU et al.   INTERNATIONAL JOURNAL OF MOLECULAR MEDICINE

Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   MUSCLE & NERVE

Targeted Myostatin Gene Editing in Multiple Mammalian Species Directed by a Single Pair of TALE Nucleases

(2013) Li Xu et al.   Molecular Therapy-Nucleic Acids

Evidence-based path to newborn screening for duchenne muscular dystrophy

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

Cardiac phenotype of Duchenne Muscular Dystrophy: Insights from cellular studies

(2012) Natalia Shirokova et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Cardiomyopathy of duchenne muscular dystrophy: Current understanding and future directions

(2011) Christopher F. Spurney   MUSCLE & NERVE

An intronic LINE-1 element insertion in the dystrophin gene aborts dystrophin expression and results in Duchenne-like muscular dystrophy in the corgi breed

(2010) Bruce F Smith et al.   LABORATORY INVESTIGATION

Preclinical drug trials in themdxmouse: Assessment of reliable and sensitive outcome measures

(2009) Christopher F. Spurney et al.   MUSCLE & NERVE

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy

(2009) Craig M. McDonald et al.   MUSCLE & NERVE