Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs.

Human molecular genetics - 15 Aug 2010

Kayali Refik, Bury Frederic, Ballard McIver, Bertoni Carmen

Abstract excerpt

Permanent correction of gene defects is an appealing approach to the treatment of genetic disorders. The use of single-stranded oligodeoxynucleotides (ssODNs) has been demonstrated to induce single-point mutations in the dystrophin gene and to restore dystrophin expression in the skeletal muscle of models of Duchenne muscular dystrophy (DMD). Here we show that ssODNs made of peptide nucleic acids (PNA-ssODNs) can...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to DNA, Single-Stranded
Share to Dystrophin
Share to Injections, Intramuscular

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Mice, Inbred mdx

Share to Muscles

Share to Myoblasts

Share to Oligodeoxyribonucleotides

Share to Peptide Nucleic Acids

Source

Identifiers

PMID: 20542988
DOI: 10.1093/hmg/ddq235

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs.PMID 20542988Read ↗

Strand bias in oligonucleotide-mediated dystrophin gene editing.Semantic proximity Read ↗

Genetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice.Semantic proximity Read ↗

Genetic therapeutic approaches for Duchenne muscular dystrophy.Semantic proximity Read ↗

Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors.Semantic proximity Read ↗

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.Semantic proximity Read ↗

Oligonucleotide-mediated gene therapy for muscular dystrophies.Semantic proximity Read ↗

New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Semantic proximity Read ↗

Molecular therapies for Duchenne muscular dystrophy.Semantic proximity Read ↗

Genetic treatments in muscular dystrophies.Semantic proximity Read ↗

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations.Semantic proximity Read ↗

Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMDSemantic proximity Read ↗

<i>In vivo</i> base editing via single myotrophic adeno-associated viruses in dystrophic mouse muscle and satellite cellsSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.