Evidence map›Paper›PMID 34781633›Full record

ArticleJournal of movement disorders2022

Gene Therapy for Huntington's Disease: The Final Strategy for a Cure?

Seulgi Byun, Mijung Lee, Manho Kim

Abstract read
In one paragraph

Article in Journal of movement disorders, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing 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

18 citing papers in PubMed.

  1. Review
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  12. Emerging Perspectives on Prime Editor Delivery to the Brain.Pharmaceuticals (Basel, Switzerland) · 2024
    Review
  13. Optimizing Screening for Intrastriatal Interventions in Huntington's Disease Using Predictive Models.Movement disorders : official journal of the Movement Disorder Society · 2024
    Article
  14. Review
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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

3 authors.

Seulgi ByunDepartment of Neurology, Seoul National University Hospital, Seoul, Korea.
Mijung LeeDepartment of Neurology, Seoul National University Hospital, Seoul, Korea.
Manho KimDepartment of Neurology, Seoul National University Hospital, Seoul, Korea.

Funding

Korea Brain Research Institute 19-BR-02-01Ministry of Science and ICTNational Research Foundation of Korea 2016M3C7A1914002
6 · The paper itself

Abstract

Huntington's disease (HD) has become a target of the first clinical trials for gene therapy among movement disorders with a genetic origin. More than 100 clinical trials regarding HD have been tried, but all failed, although there were some improvements limited to symptomatic support. Compared to other neurogenetic disorders, HD is known to have a single genetic target. Thus, this is an advantage and its cure is more feasible than any other movement disorder with heterogeneous genetic causes. In this review paper, the authors attempt to cover the characteristics of HD itself while providing an overview of the gene transfer methods currently being researched, and will introduce an experimental trial with a preclinical model of HD followed by an update on the ongoing clinical trials for patients with HD.

Indexed as

Gene transferHuntington’s diseaseMovement disorder

Identifiers

PMID34781633
PMCPMC8820879

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.