Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice

Casimersen: First Approval

(2021) Matt Shirley   DRUGS

Golodirsen: First Approval

(2020) Young-A Heo   DRUGS

Viltolarsen: First Approval

(2020) Sohita Dhillon   DRUGS

Premature termination codons in theDMDgene cause reduced local mRNA synthesis

(2020) Raquel García-Rodríguez et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

(2019) Maria K. Tsoumpra et al.   EBioMedicine

CRISPR-Cas9 screens identify regulators of antibody–drug conjugate toxicity

(2019) C. Kimberly Tsui et al.   Nature Chemical Biology

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

Eteplirsen in the treatment of Duchenne muscular dystrophy

(2017) Kenji Rowel Lim et al.   Drug Design Development and Therapy

Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles

(2016) Tsukasa Sugo et al.   JOURNAL OF CONTROLLED RELEASE

Identifying Non–Duchenne Muscular Dystrophy–Positive and False Negative Results in Prior Duchenne Muscular Dystrophy Newborn Screening Programs

(2016) Michele A. Gatheridge et al.   JAMA Neurology

Antibody Drug Conjugates: Design and Selection of Linker, Payload and Conjugation Chemistry

(2015) Jessica R. McCombs et al.   AAPS Journal

Exon skipping therapy for Duchenne muscular dystrophy

(2015) Ryszard Kole et al.   ADVANCED DRUG DELIVERY REVIEWS

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides

(2015) Umar Burki et al.   Nucleic Acid Therapeutics

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

(2015) Corinne A. Betts et al.   Scientific Reports

Metabolic Catastrophe in Mice Lacking Transferrin Receptor in Muscle

(2015) Tomasa Barrientos et al.   EBioMedicine

Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx Mice

(2014) Xianjun Gao et al.   MOLECULAR THERAPY

Mutation Types and Aging Differently Affect Revertant Fiber Expansion in Dystrophic Mdx and Mdx52 Mice

(2013) Yusuke Echigoya et al.   PLoS One

Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges

(2012) Virginia Arechavala-Gomeza et al.   CURRENT GENE THERAPY

NIH Image to ImageJ: 25 years of image analysis

(2012) Caroline A Schneider et al.   NATURE METHODS

Chemical and Mechanistic Toxicology Evaluation of Exon Skipping Phosphorodiamidate Morpholino Oligomers in mdx Mice

(2011) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Accurate quantification of dystrophin mRNA and exon skipping levels in Duchenne Muscular Dystrophy

(2010) Pietro Spitali et al.   LABORATORY INVESTIGATION

Laminins

(2009) Madeleine Durbeej   CELL AND TISSUE RESEARCH

Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort

(2009) Kevin M. Flanigan et al.   HUMAN MUTATION

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

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

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY