Evidence map›Paper›PMID 39027419›Full record

ArticleMolecular therapy. Nucleic acids2024

Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant

Naoki Iwamoto, Yuanjing Liu, Maria Frank-Kamenetsky, Abbie Maguire, Wei Chou Tseng, Kristin Taborn, Nayantara Kothari, Ali Akhtar, Keith Bowman, Juili Dilip Shelke and 23 more

Abstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
–field-weighted citation impact
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

13 citing papers in PubMed.

  1. Article
  2. Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  3. Review
  4. Review
  5. Article
  6. Review
  7. Allele-selective von Willebrand factor silencing.Research and practice in thrombosis and haemostasis · 2026
    Article
  8. Development of Antisense Gapmers for the Treatment of Huntington's Disease.Methods in molecular biology (Clifton, N.J.) · 2026
    Article
  9. Article
  10. Article
  11. Article
  12. Review
  13. Review
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

33 authors.

Naoki IwamotoWave Life Sciences, Cambridge, MA 02138, USA.
Yuanjing LiuWave Life Sciences, Cambridge, MA 02138, USA.
Maria Frank-KamenetskyWave Life Sciences, Cambridge, MA 02138, USA.
Abbie MaguireWave Life Sciences, Cambridge, MA 02138, USA.
Wei Chou TsengWave Life Sciences, Cambridge, MA 02138, USA.
Kristin TabornWave Life Sciences, Cambridge, MA 02138, USA.
Nayantara KothariWave Life Sciences, Cambridge, MA 02138, USA.
Ali AkhtarWave Life Sciences, Cambridge, MA 02138, USA.
Keith BowmanWave Life Sciences, Cambridge, MA 02138, USA.
Juili Dilip ShelkeWave Life Sciences, Cambridge, MA 02138, USA.
Anthony LamattinaWave Life Sciences, Cambridge, MA 02138, USA.
Xiao Shelley HuWave Life Sciences, Cambridge, MA 02138, USA.
Hyun Gyung JangWave Life Sciences, Cambridge, MA 02138, USA.
Pachamuthu KandasamyWave Life Sciences, Cambridge, MA 02138, USA.
Fangjun LiuWave Life Sciences, Cambridge, MA 02138, USA.
Ken LongoWave Life Sciences, Cambridge, MA 02138, USA.
Richard LoobyWave Life Sciences, Cambridge, MA 02138, USA.
MeenaWave Life Sciences, Cambridge, MA 02138, USA.
Jake MettervilleWave Life Sciences, Cambridge, MA 02138, USA.
Qianli PanWave Life Sciences, Cambridge, MA 02138, USA.
Erin Purcell-EstabrookWave Life Sciences, Cambridge, MA 02138, USA.
Mamoru ShimizuWave Life Sciences, Cambridge, MA 02138, USA.
Priyanka Shiva PrakashaWave Life Sciences, Cambridge, MA 02138, USA.
Stephany StandleyWave Life Sciences, Cambridge, MA 02138, USA.
Hansini UpadhyayWave Life Sciences, Cambridge, MA 02138, USA.
Hailin YangWave Life Sciences, Cambridge, MA 02138, USA.
Yuan YinWave Life Sciences, Cambridge, MA 02138, USA.
Anderson ZhaoWave Life Sciences, Cambridge, MA 02138, USA.
Christopher FrancisWave Life Sciences, Cambridge, MA 02138, USA.
Mike ByrneWave Life Sciences, Cambridge, MA 02138, USA.
Elena DaleWave Life Sciences, Cambridge, MA 02138, USA.
Gregory L VerdineDepartment of Stem Cell and Regenerative Biology, Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
Chandra VargeeseWave Life Sciences, Cambridge, MA 02138, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Huntington's disease (HD) is an autosomal dominant disease caused by the expansion of cytosine-adenine-guanine (CAG) repeats in one copy of the

Indexed as

allele-selective silencingHTTHuntington’s diseaseMT: Oligonucleotides: Therapies and Applicationsmutant huntingtinphosphoryl guanidineSELECT-HDSNP3stereopure antisense oligonucleotidewild-type huntingtinWVE-003

Identifiers

PMID39027419
PMCPMC11255113

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.