Evidence map›Paper›PMID 41090742›Full record

ReviewCells2025

Revolutionizing Huntington's Disease Treatment: Breakthroughs in AAV-Mediated Gene Therapy.

Pedram Moeini Gavgani, Mario García-Domínguez

Abstract readReview
In one paragraph

Review in Cells, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Review
  3. 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

2 authors.

Pedram Moeini GavganiDNA & RNA Medicine Division, CIMA-Universidad de Navarra, 31008 Pamplona, Spain.
Mario García-DomínguezProgram of Immunology and Immunotherapy, CIMA-Universidad de Navarra, 31008 Pamplona, Spain.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Huntington's Disease (HD) is an inherited neurodegenerative condition caused by an expansion of CAG repeats in the Huntingtin (HTT) gene, leading to a toxic form of the HTT protein. Despite advances in understanding the disease and developing symptomatic treatments, effective therapies for modifying its progression remain limited. Among emerging and novel treatments for central nervous system (CNS) disorders, gene therapy (GT), particularly using adeno-associated virus (AAV)-mediated gene delivery, holds great promise. Numerous preclinical and clinical trials are exploring the benefits of AAVs for treating neurodegenerative and genetic diseases. However, while widely used and investigated in rare and genetic disease treatment, AAVs' potential for HD treatment remains underexplored. The absence of a comprehensive collection of previous reports, advancements, and methodologies regarding exclusively AAV-mediated GT for HD is notable and prompted us to address this gap. The current review compiles the available and emerging information regarding the application of AAVs in HD therapy, outlines the promise of this approach, and highlights the necessity of conducting further studies to achieve efficient HD treatment. The authors hope that the current review will guide further research to unlock the full potential of AAVs in treating HD.

Indexed as

DependovirusGenetic TherapyHuntington DiseaseAnimalsGenetic VectorsHumansHuntingtin ProteinHuntingtin Proteinadeno-associated viral vectorgene therapyhuntingtin geneHuntington’s diseaseneurodegenerative disorders

Identifiers

PMID41090742
PMCPMC12523392

What OpenQuestion holds

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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.