Evidence map›Paper›PMID 30093643›Full record

ReviewNature reviews. Drug discovery2018

Gene therapy for neurological disorders: progress and prospects.

Benjamin E Deverman, Bernard M Ravina, Krystof S Bankiewicz, Steven M Paul, Dinah W Y Sah

Erratum issuedAbstract readReview
PubMed Publisher
In one paragraph

Review in Nature reviews. Drug discovery, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 180 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
180citing papers in PubMed, 1 pooled it
13.6field-weighted citation impact, top 1% of its field
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

180 citing papers in PubMed, 1 synthesis or guideline pooled it, 295 citations in OpenAlex.

  1. Pooled it
  2. Delta power robustly predicts cognitive function in Angelman syndrome.Annals of clinical and translational neurology · 2021
    Trial
  3. Trial
  4. Article
  5. Review
  6. Article
  7. Article
  8. Article
  9. Review
  10. Article
  11. Review
  12. Article
  13. Review
  14. Review
  15. Article
  16. Genetic therapies for neurological diseases.Pharmacological reviews · 2026
    Review
  17. Review
  18. Review
  19. Review
  20. Developing a minimally invasive gene therapy for multiple sclerosis.Molecular therapy. Methods & clinical development · 2025
    Article

120 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

5 authors at 3 institutions in 1 country.

Benjamin E DevermanDivision of Biology and Biological Engineering, California Institute of Technology, Pasadena, CA, USA.
Bernard M RavinaVoyager Therapeutics, Cambridge, MA, USA.
Krystof S BankiewiczDepartment of Neurological Surgery, University of California-San Francisco, San Francisco, CA, USA.
Steven M PaulVoyager Therapeutics, Cambridge, MA, USA.
Dinah W Y SahVoyager Therapeutics, Cambridge, MA, USA.
Voyager Therapeutics (United States) · USBroad Institute · USNeurological Surgery · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic proteins, microRNAs, antibodies or gene-editing machinery have been successfully delivered to the central nervous system with natural or engineered viral capsids via various routes of administration. Importantly, initial clinical studies have demonstrated encouraging safety and efficacy in diseases such as Parkinson disease and spinal muscular atrophy, as well as durability of transgene expression. Here, we discuss key considerations and challenges in the future design and development of therapeutic AAV vectors, highlighting the most promising targets and recent clinical advances.

Identifiers

PMID30093643
OpenAlexW4233394827

What OpenQuestion holds

Textmetadata
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.