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

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Genome-wide identification of CRISPR/Cas9 off-targets in human genome

(2014) Jinzhi Duan et al.   CELL RESEARCH

Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells

(2014) Xuebing Wu et al.   NATURE BIOTECHNOLOGY

Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease

(2014) Cem Kuscu et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Characterization of 65 Epitope-Specific Dystrophin Monoclonal Antibodies in Canine and Murine Models of Duchenne Muscular Dystrophy by Immunostaining and Western Blot

(2014) Kasun Kodippili et al.   PLoS One

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering

(2013) Hui Yang et al.   CELL

Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

(2013) David G Ousterout et al.   MOLECULAR THERAPY

TALEN-based Gene Correction for Epidermolysis Bullosa

(2013) Mark J Osborn et al.   MOLECULAR THERAPY

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

(2013) Lior Zangi et al.   NATURE BIOTECHNOLOGY

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

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

Satellite Cells and the Muscle Stem Cell Niche

(2013) Hang Yin et al.   PHYSIOLOGICAL REVIEWS

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

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

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

(2012) M. Jinek et al.   SCIENCE

Satellite cells, connective tissue fibroblasts and their interactions are crucial for muscle regeneration

(2011) M. M. Murphy et al.   DEVELOPMENT

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

Tissue-specific gene delivery via nanoparticle coating

(2009) Todd J. Harris et al.   BIOMATERIALS