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

Translational and Regulatory Challenges for Exon Skipping Therapies

3 Sept 2014

Abstract excerpt

Several translational challenges are currently impeding the therapeutic development of antisense-mediated exon skipping approaches for rare diseases. Some of these are inherent to developing therapies for rare diseases, such as small patient numbers and limited information on natural history and interpretation of appropriate clinical outcome measures. Others are inherent to the antisense oligonucleotide...

Read the complete abstract on PubMed

Topics

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

Share to CRISPR and Genetic Engineering
Share to Neurogenetic and Muscular Disorders Research
Share to Pluripotent Stem Cells Research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 25184444
DOI: 10.1089/hum.2014.086

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Translational and Regulatory Challenges for Exon Skipping TherapiesPMID 25184444Read ↗

Exon skipping: a first in class strategy for Duchenne muscular dystrophySemantic proximity Read ↗

194th ENMC international workshop. 3rd ENMC workshop on exon skipping: Towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8–10 December 2012, Naarden, The NetherlandsSemantic proximity Read ↗

Overview on applications of antisense-mediated exon skipping.Semantic proximity Read ↗

Translational development of splice-modifying antisense oligomersSemantic proximity Read ↗

Antisense-mediated exon skipping: Taking advantage of a trick from Mother Nature to treat rare genetic diseasesSemantic proximity Read ↗

Antisense Oligonucleotide-Mediated Exon-skipping Therapies: Precision Medicine Spreading from Duchenne Muscular DystrophySemantic proximity Read ↗

A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground.Semantic proximity Read ↗

Antisense-mediated splice intervention to treat human disease: the odyssey continuesSemantic proximity Read ↗

Delivery is key: lessons learnt from developing splice‐switching antisense therapiesSemantic proximity Read ↗

Clinical applications of exon-skipping antisense oligonucleotides in neuromuscular diseasesSemantic proximity Read ↗

Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments.Semantic proximity Read ↗

Expansion of Splice-Switching Therapy with Antisense OligonucleotidesSemantic proximity Read ↗

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