Evidence map›Paper›PMID 41389437›Full record

ReviewPharmacological reviews2026

Genetic therapies for neurological diseases.

Ahad A Rahim, Manju A Kurian, Haiyan Zhou, Ross Ferguson, Sarah J Tabrizi, Gabriele Lignani, Kristian Aquilina, Simon N Waddington

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Pharmacological reviews, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

8 authors.

Ahad A RahimDepartment of Pharmacology, School of Pharmacy, University College London, London, United Kingdom. Electronic address: a.rahim@ucl.ac.uk.
Manju A KurianDevelopmental Neurosciences, Zayed Centre for Research into Rare Disease in Children, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom; Department of Neurology, Great Ormond Street Hospital for Children, London, United Kingdom.
Haiyan ZhouGenetics and Genomic Medicine Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom; National Institute for Health and Care Research, Great Ormond Street Hospital Biomedical Research Center, London, United Kingdom.
Ross FergusonHuntington's Disease Centre, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom; Dementia Research Institute at University College London, London, United Kingdom.
Sarah J TabriziHuntington's Disease Centre, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom; Dementia Research Institute at University College London, London, United Kingdom.
Gabriele LignaniResearch Department of Epilepsy, Queen Square Institute of Neurology, University College London, London, United Kingdom.
Kristian AquilinaDepartment of Neurosurgery, Great Ormond Street Hospital, London, United Kingdom.
Simon N WaddingtonDepartment of Maternal and Fetal Medicine, Elizabeth Garrett Anderson Institute for Women's Health, University College London, London, United Kingdom. Electronic address: s.waddington@ucl.ac.uk.

Funding

Wellcome Trust 204841/Z/16/ZWellcome Trust 223082/Z/21/Z
6 · The paper itself

Abstract

Often, gene therapy reviews concentrate upon specific therapeutic modalities-particularly either viral vector-mediated or a nonviral approach. Here, we draw together a comprehensive array of knowledge across the field of genetic therapy for genetic neurological disease. The sections on preclinical and clinical application of viral vectors are followed by sections on RNA-based therapies and then by antisense oligonucleotide approaches also in preclinical and clinical settings. We present a separate section on gene editing strategies and conclude with a section elaborating on the neurosurgical techniques and the expertise required for clinical application of many of these technologies. SIGNIFICANCE STATEMENT: Genetic therapies have significant potential to treat life-limiting neurological diseases. This review examines the different approaches, clinical successes, and considerations on how to deploy them.

Indexed as

Genetic TherapyNervous System DiseasesAnimalsGene EditingGenetic VectorsHumansOligonucleotides, AntisenseOligonucleotides, Antisense

Identifiers

PMID41389437
PMCPMC12881687

What Socratic 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 Socratic graph.