Evidence map›Paper›PMID 33863599›Full record

ReviewTrends in pharmacological sciences2021

Delivering AAV to the Central Nervous and Sensory Systems.

Cole W Peters, Casey A Maguire, Killian S Hanlon

Abstract readReview
In one paragraph

Review in Trends in pharmacological sciences, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.

0numbers the graph read from it
0cells of the map it votes in
29citing 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

29 citing papers in PubMed.

  1. Article
  2. Review
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  7. Review
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  12. Review
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  16. Review
  17. Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain.Molecular therapy. Methods & clinical development · 2023
    Article
  18. Article
  19. Review
  20. 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

3 authors.

Cole W PetersDepartment of Neurobiology, Harvard Medical School, Boston, MA 02115, USA.
Casey A MaguireMolecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02115, USA.
Killian S HanlonDepartment of Neurobiology, Harvard Medical School, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Department of Neurology, Massachusetts General Hospital, Charlestown, MA 02129, USA. Electronic address: killian_hanlon@hms.harvard.edu.

Funding

Enhanced gene delivery for CNS and sensory disordersR01DC017117 · NIDCD · MASSACHUSETTS GENERAL HOSPITAL · PI MAGUIRE, CASEY A · 2019 to 2023
$1.8M
NIDCD NIH HHS R01 DC017117
6 · The paper itself

Abstract

As gene therapy enters mainstream medicine, it is more important than ever to have a grasp of exactly how to leverage it for maximum benefit. The development of new targeting strategies and tools makes treating patients with genetic diseases possible. Many Mendelian disorders are amenable to gene replacement or correction. These often affect post-mitotic tissues, meaning that a single stably expressing therapy can be applied. Recent years have seen the development of a large number of novel viral vectors for delivering specific therapies. These new vectors - predominately recombinant adeno-associated virus (AAV) variants - target nervous tissues with differing efficiencies. This review gives an overview of current gene therapies in the brain, ear, and eye, and describes the optimal approaches, depending on cell type and transgene. Overall, this work aims to serve as a primer for gene therapy in the central nervous and sensory systems.

Indexed as

DependovirusNerve TissueGenetic TherapyGenetic VectorsGene Transfer TechniquesHumansTransgenesAAVbraingene therapyinner earretina

Identifiers

PMID33863599
PMCPMC9302199

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.