Evidence map›Paper›PMID 32963936›Full record

ReviewActa pharmaceutica Sinica. B2020

Gene therapy for neurodegenerative disorders: advances, insights and prospects.

Wei Chen, Yang Hu, Dianwen Ju

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Acta pharmaceutica Sinica. B, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 83 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
83citing papers in PubMed, 1 pooled it
–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

83 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Observational
  4. Article
  5. Review
  6. Lipid and polymeric nanocarriers for siRNA delivery to the brain.Journal of controlled release : official journal of the Controlled Release Society · 2025
    Review
  7. Article
  8. Article
  9. Nanotherapy for Neural Retinal Regeneration.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  10. Engineering the Immune Response to Biomaterials.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  11. Review
  12. Review
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23 more citing papers are in PubMed but not listed here.

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.

Wei ChenDepartment of Biological Medicines, Fudan University School of Pharmacy, Shanghai 201203, China.
Yang HuDepartment of Ophthalmology, Stanford University School of Medicine, Palo Alto, CA 94304, USA.
Dianwen JuDepartment of Biological Medicines, Fudan University School of Pharmacy, Shanghai 201203, China.

Funding

Elucidating Neuron-Intrinsic Molecular Mechanisms of Optic Nerve RegenerationR01EY024932 · NEI · TEMPLE UNIV OF THE COMMONWEALTH · PI Yang Hu · 2015 to 2026
$4.8M
Neuroprotection by Modulating ER Stress in GlaucomaR01EY023295 · NEI · TEMPLE UNIV OF THE COMMONWEALTH · PI HU, YANG · 2013 to 2022
$3.8M
Developing Novel Neuroprotective Strategies for EAE/Optic NeuritisR01EY028106 · NEI · STANFORD UNIVERSITY · PI HU, YANG · 2018 to 2021
$1.9M
Development and Characterization of Silicone Oil-Induced Reversible Ocular Hypertension Glaucoma Model R21EY031063 · NEI · STANFORD UNIVERSITY · PI HU, YANG · 2019 to 2019
$324k
NEI NIH HHS R01 EY023295NEI NIH HHS R01 EY024932NEI NIH HHS R01 EY028106NEI NIH HHS R21 EY031063
6 · The paper itself

Abstract

Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) and Huntington's disease (HD). Some early clinical trials have failed to achieve satisfactory therapeutic effects. Efforts to enhance effectiveness are now concentrating on three major fields: identification of new vectors, novel therapeutic targets, and reliable of delivery routes for transgenes. These approaches are being assessed closely in preclinical and clinical trials, which may ultimately provide powerful treatments for patients. Here, we discuss advances and challenges of gene therapy for neurodegenerative disorders, highlighting promising technologies, targets, and future prospects.

Indexed as

AADC, aromatic-l-amino-acidAAVs, adeno-associated virusesAD, Alzheimer's diseaseAdeno-associated virusesAdv, adenovirusARSA, arylsulfatase AASOs, antisense oligonucleotidesASPA, aspartoacylaseBBB, blood–brain barrierBCSFB, blood–cerebrospinal fluid barrierBip, glucose regulated protein 78BRB, blood–retina barrierCentral nervous systemCHOP, CCAAT/enhancer binding homologous proteinCLN6, ceroidlipofuscinosis neuronal protein 6CNS, central nervous systemCSF, cerebrospinal fluidDelivery routesER, endoplasmic reticulumFDA, U.S. Food and Drug AdministrationGAA, lysosomal acid α-glucosidaseGAD, glutamic acid decarboxylaseGDNF, glial derived neurotrophic factorGene therapyHD, Huntington's diseaseHSPGs, heparin sulfate proteoglycansHTT, mutant huntingtinIDS, iduronate 2-sulfataseLamp2a, lysosomal-associated membrane protein 2aLVs, retrovirus/lentivirusmTOR, mammalian target of rapamycinNeurodegenerative disordersNGF, nerve growth factorPD, Parkinson's diseasePGRN, ProgranulinPINK1, putative kinase 1PTEN, phosphatase and tensin homologRGCs, retinal ganglion cellsRNAi, RNA interferenceRPE, retinal pigmented epithelialSGSH, lysosomal heparan-N-sulfamidase genesiRNA, small interfering RNASMN, survival motor neuronSOD, superoxide dismutaseSUMF, sulfatase-modifying factorTFEB, transcription factor EBTPP1, tripeptidyl peptidase 1TREM2, triggering receptor expressed on myeloid cells 2UPR, unfolded protein responseZFPs, zinc finger proteins

Identifiers

PMID32963936
PMCPMC7488363

What OpenQuestion holds

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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.