CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

Cpf1 nucleases demonstrate robust activity to induce DNA modification by exploiting homology directed repair pathways in mammalian cells

(2016) Eszter Tóth et al.   Biology Direct

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

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

(2016) Li Xu et al.   MOLECULAR THERAPY

The CRISPR-associated DNA-cleaving enzyme Cpf1 also processes precursor CRISPR RNA

(2016) Ines Fonfara et al.   NATURE

Generation of knockout mice by Cpf1-mediated gene targeting

(2016) Yongsub Kim et al.   NATURE BIOTECHNOLOGY

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

(2016) M. A. DeWitt et al.   Science Translational Medicine

Genome Editing of Monogenic Neuromuscular Diseases

(2016) Chengzu Long et al.   JAMA Neurology

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Viral vector-mediated gene therapies

(2015) Katrin Hollinger et al.   CURRENT OPINION IN NEUROLOGY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

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

MED13-dependent signaling from the heart confers leanness by enhancing metabolism in adipose tissue and liver

(2014) K. K. Baskin et al.   EMBO Molecular Medicine

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Chemically defined generation of human cardiomyocytes

(2014) Paul W Burridge et al.   NATURE METHODS

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

(2014) C. Long et al.   SCIENCE

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

(2013) Yuxuan Wu et al.   Cell Stem Cell

Role of telomere dysfunction in cardiac failure in Duchenne muscular dystrophy

(2013) Foteini Mourkioti et al.   NATURE CELL BIOLOGY

Obligate Ligation-Gated Recombination (ObLiGaRe): Custom-designed nuclease-mediated targeted integration through nonhomologous end joining

(2012) M. Maresca et al.   GENOME RESEARCH

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

(2012) M. Jinek et al.   SCIENCE

New connections between splicing and human disease

(2012) Richard A. Padgett   TRENDS IN GENETICS

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice

(2011) Brian Bostick et al.   MOLECULAR THERAPY

Total Skeletal Muscle PGC-1 Deficiency Uncouples Mitochondrial Derangements from Fiber Type Determination and Insulin Sensitivity

(2010) Christoph Zechner et al.   Cell Metabolism

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Genetic and pharmacologic inhibition of mitochondrial-dependent necrosis attenuates muscular dystrophy

(2008) Douglas P Millay et al.   NATURE MEDICINE