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

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1

31 Jan 2023

Abstract excerpt

Myotonic dystrophy type 1 (DM1) is one of the most common muscular dystrophies and can be potentially treated with antisense therapy decreasing mutant DMPK, targeting miRNAs or their binding sites or via a blocking mechanism for MBNL1 displacement from the repeats. Unconjugated antisense molecules are able to correct the disease phenotype in mouse models, but they show poor muscle penetration upon systemic...

Read the complete abstract on PubMed

Topics

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

Share to Cardiomyopathy and Myosin Studies
Share to Genetic Neurodegenerative Diseases
Share to Muscle Physiology and Disorders

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 36769018
DOI: 10.3390/ijms24032697

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1PMID 36769018Read ↗

Bottlebrush Polymer Conjugates for Enhanced Antisense Oligonucleotide Therapy in Myotonic Dystrophy Type 1Semantic proximity Read ↗

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseasesSemantic proximity Read ↗

Antisense Oligonucleotide-Based Therapy for Neuromuscular DiseaseSemantic proximity Read ↗

Targeting nuclear RNA for in vivo correction of myotonic dystrophy.Semantic proximity Read ↗

Therapeutics development in myotonic dystrophy type 1Semantic proximity Read ↗

Recent Trends in Antisense Therapies for Duchenne muscular dystrophySemantic proximity Read ↗

Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1Semantic proximity Read ↗

Identification and Characterization of Modified Antisense Oligonucleotides Targeting DMPK in Mice and Nonhuman Primates for the Treatment of Myotonic Dystrophy Type 1Semantic proximity Read ↗

Challenges and future perspective of antisense therapy for spinal muscular atrophy: A reviewSemantic proximity Read ↗

The bench to bedside journey of tricyclo-DNA antisense oligonucleotides for the treatment of Duchenne muscular dystrophySemantic proximity Read ↗

Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseasesSemantic proximity Read ↗

Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse modelSemantic proximity Read ↗

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