Evidence map›Paper›PMID 29271929›Full record

ReviewBiomedicines2017

The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders.

Philippine Aupy, Lucía Echevarría, Karima Relizani, Aurélie Goyenvalle

Open access · goldAbstract readReview
In one paragraph

Review in Biomedicines, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.9field-weighted citation impact, top 23% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 22 citations in OpenAlex.

  1. Review
  2. Review
  3. Article
  4. Review
  5. Review
  6. Review
  7. Review
  8. Review
  9. Review
  10. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 1 institution in 1 country.

Philippine AupyINSERM U1179, UFR des Sciences de la Santé, University of Versailles St-Quentin, 78180 Montigny le Bretonneux, France. philippine.aupy2@uvsq.fr.
Lucía EchevarríaINSERM U1179, UFR des Sciences de la Santé, University of Versailles St-Quentin, 78180 Montigny le Bretonneux, France. lucia.echevarria-zamora@uvsq.fr.
Karima RelizaniINSERM U1179, UFR des Sciences de la Santé, University of Versailles St-Quentin, 78180 Montigny le Bretonneux, France. sonia.relizani-karima@uvsq.fr.
Aurélie GoyenvalleINSERM U1179, UFR des Sciences de la Santé, University of Versailles St-Quentin, 78180 Montigny le Bretonneux, France. aurelie.goyenvalle@uvsq.fr.ORCID 0000-0002-1281-4752
Inserm · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with promising developments of more potent and nuclease resistant chemistries. Despite these recent clinical successes and advances in chemistry and design, effective delivery of ASOs to their target tissues remains a major issue. This review will describe the latest advances obtained with the tricyclo-DNA (tcDNA) chemistry which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration. We will examine the variety of therapeutic approaches using both fully modified tcDNA-ASOs and gapmers, including splice switching applications, correction of aberrant splicing, steric blocking strategies and targeted gene knock-down mediated by RNase H recruitment. We will then discuss the merits and potential liabilities of the tcDNA chemistry in the context of ASO drug development.

Indexed as

antisense oligonucleotidesdeliveryexon-re-inclusionexon-skippinggapmerssplice-switchingtricyclo-DNA

Identifiers

PMID29271929
PMCPMC5874659
OpenAlexW2781370412

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.