Evidence map›Paper›PMID 39985571›Full record

ReviewJournal of neurology2025

Genetic therapies for movement disorders - current status.

J Sartorelli, J Ng, A A Rahim, S N Waddington, M A Kurian

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Journal of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Review
  2. 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

5 authors.

J SartorelliDevelopmental Neurosciences, Zayed Centre for Research Into Rare Disease in Children, UCL Great Ormond Street Institute of Child Health, 20 Guilford Street, London, WC1N 1DZ, UK.
J NgGenetic Therapy Accelerator Centre, UCL Queen Square Institute of Neurology, London, UK.
A A RahimUCL School of Pharmacy, University College London, London, UK.
S N WaddingtonGene Transfer Technology Group, Institute for Women's Health, University College London, London, UK.
M A KurianDevelopmental Neurosciences, Zayed Centre for Research Into Rare Disease in Children, UCL Great Ormond Street Institute of Child Health, 20 Guilford Street, London, WC1N 1DZ, UK. manju.kurian@ucl.ac.uk.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Movement disorders are a group of heterogeneous neurological conditions associated with alterations of tone, posture and voluntary movement. They may either occur in isolation or as part of a multisystemic condition. More recently, the advent of next generation sequencing technologies has facilitated better understanding of the underlying causative genes and molecular pathways, thereby identifying targets for genetic therapy. In this review, we summarize the advances in genetic therapy approaches for both hyperkinetic and hypokinetic movement disorders, including Parkinson's Disease, Huntington's Disease and rarer monogenic conditions of childhood onset. While there have been significant advances in the field, multiple challenges remain, related to safety, toxicity, efficacy and brain biodistribution, which will need to be addressed by the next generation of genetic therapies.

Indexed as

Genetic TherapyMovement DisordersHumansHuntington DiseaseBrainGeneGenetic therapyMovement disorderViral vector

Identifiers

PMID39985571
PMCPMC11846774

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.