Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

(2012) Aurélie Goyenvalle et al.   HUMAN MOLECULAR GENETICS

Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases

(2012) Fayza Daboussi et al.   NUCLEIC ACIDS RESEARCH

Inhibition of Non-Homologous End Joining Repair Impairs Pancreatic Cancer Growth and Enhances Radiation Response

(2012) Ying-Hua Li et al.   PLoS One

Genome Engineering With Zinc-Finger Nucleases

(2011) Dana Carroll   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

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Context dependence between subdomains in the DNA binding interface of the I-CreI homing endonuclease

(2011) Sylvestre Grizot et al.   NUCLEIC ACIDS RESEARCH

Therapeutic Targeting of the DNA Mismatch Repair Pathway

(2010) S. A. Martin et al.   CLINICAL CANCER RESEARCH

Mechanisms and regulation of DNA end resection

(2010) Maria Pia Longhese et al.   EMBO JOURNAL

Meganucleases can restore the reading frame of a mutated dystrophin

(2010) P Chapdelaine et al.   GENE THERAPY

Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials

(2010) Linda J. Popplewell et al.   NEUROMUSCULAR DISORDERS

Immortalized Skin Fibroblasts Expressing Conditional MyoD as a Renewable and Reliable Source of Converted Human Muscle Cells to Assess Therapeutic Strategies for Muscular Dystrophies: Validation of an Exon-Skipping Approach to Restore Dystrophin in Duchenne Muscular Dystrophy Cells

(2009) Soraya Chaouch et al.   HUMAN GENE THERAPY

Lentiviral-mediated Genetic Correction of Hematopoietic and Mesenchymal Progenitor Cells From Fanconi Anemia Patients

(2009) Ariana Jacome et al.   MOLECULAR THERAPY

Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease

(2009) Sylvestre Grizot et al.   NUCLEIC ACIDS RESEARCH

Identification of an Intronic Splicing Enhancer Essential for the Inclusion ofFGFR2Exon IIIc

(2008) Puneet Seth et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY