Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum

Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases

(2010) Liutao Du et al.   JOURNAL OF IMMUNOLOGICAL METHODS

Naturally- and experimentally-designed restorations of the Parkin gene deficit in autosomal recessive juvenile parkinsonism

(2009) Hirohide Asai et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Cellular Uptake of Neutral Phosphorodiamidate Morpholino Oligomers

(2009) Patrick Iversen et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

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

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

Delivery of nucleic acids with a stearylated (RxR)4 peptide using a non-covalent co-incubation strategy

(2009) Taavi Lehto et al.   JOURNAL OF CONTROLLED RELEASE

Nonaminoglycoside compounds induce readthrough of nonsense mutations

(2009) Liutao Du et al.   JOURNAL OF EXPERIMENTAL MEDICINE

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

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Arginine-rich cell penetrating peptides: Design, structure–activity, and applications to alter pre-mRNA splicing by steric-block oligonucleotides

(2008) R. Abes et al.   JOURNAL OF PEPTIDE SCIENCE

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

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY

Ataxia-telangiectasia: from a rare disorder to a paradigm for cell signalling and cancer

(2008) Martin F. Lavin   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

Cell penetrating peptide conjugates of steric block oligonucleotides

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