Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers†

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

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

Therapeutic Potential of Splice-Switching Oligonucleotides

(2009) John Bauman et al.   OLIGONUCLEOTIDES

Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: A comparative study from the Pediatric Cardiomyopathy Registry

(2008) David M. Connuck et al.   AMERICAN HEART JOURNAL

Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance

(2008) G. M. Buyse et al.   EUROPEAN HEART JOURNAL

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

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

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

(2008) Brian Bostick et al.   HUMAN GENE THERAPY

Peptide-Conjugated Antisense Therapy Takes a Skip Ahead

(2008) Dominic J Wells   MOLECULAR THERAPY

Emergent Dilated Cardiomyopathy Caused by Targeted Repair of Dystrophic Skeletal Muscle

(2008) DeWayne Townsend et al.   MOLECULAR THERAPY

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

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

(2008) B. Wu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hypertrophic cardiomyopathy: the genetic determinants of clinical disease expression

(2008) Andre Keren et al.   Nature clinical practice. Cardiovascular medicine

Cell penetrating peptide conjugates of steric block oligonucleotides

(2007) Bernard Lebleu et al.   ADVANCED DRUG DELIVERY REVIEWS