Exon Skipping and Gene Transfer Restore Dystrophin Expression in Human Induced Pluripotent Stem Cells-Cardiomyocytes Harboring DMD Mutations

Drug evaluation in cardiomyocytes derived from human induced pluripotent stem cells carrying a long QT syndrome type 2 mutation

(2011) Elena Matsa et al.   EUROPEAN HEART JOURNAL

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

Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

(2011) HaiFang Yin et al.   MOLECULAR THERAPY

Faster generation of hiPSCs by coupling high-titer lentivirus and column-based positive selection

(2011) Emily Dick et al.   Nature Protocols

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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

PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation

(2011) Kariem Ezzat et al.   NUCLEIC ACIDS RESEARCH

Long-Term Systemic Administration of Unconjugated Morpholino Oligomers for Therapeutic Expression of Dystrophin by Exon Skipping in Skeletal Muscle: Implications for Cardiac Muscle Integrity

(2011) Alberto Malerba et al.   Nucleic Acid Therapeutics

New insights in gene-derived therapy: the example of Duchenne muscular dystrophy

(2010) Annemieke Aartsma-Rus et al.   Annals of the New York Academy of Sciences

Evaluating the utility of cardiomyocytes from human pluripotent stem cells for drug screening

(2010) Emily Dick et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

RNA-targeted splice-correction therapy for neuromuscular disease

(2010) M. J. A. Wood et al.   BRAIN

Molecular Analyses of Human Induced Pluripotent Stem Cells and Embryonic Stem Cells

(2010) Mark H. Chin et al.   Cell Stem Cell

Modulation of human embryonic stem cell-derived cardiomyocyte growth: A testbed for studying human cardiac hypertrophy?

(2010) Gábor Földes et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

(2010) Qi-Long Lu et al.   MOLECULAR THERAPY

Progress in muscular dystrophy research with special emphasis on gene therapy

(2010) Hideo SUGITA et al.   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy

(2010) Annemieke Aartsma-Rus   RNA Biology

Two new protocols to enhance the production and isolation of human induced pluripotent stem cell lines

(2010) Emily Dick et al.   Stem Cell Research

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

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

Guidelines and Techniques for the Generation of Induced Pluripotent Stem Cells

(2008) Nimet Maherali et al.   Cell Stem Cell

Long-Term Benefit of Adeno-Associated Virus/Antisense-Mediated Exon Skipping in Dystrophic Mice

(2008) Michela Alessandra Denti et al.   HUMAN GENE THERAPY