Evidence map›Paper›PMID 33526943›Full record

ReviewNature neuroscience2021

Gene-based therapies for neurodegenerative diseases.

Jichao Sun, Subhojit Roy

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Nature neuroscience, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 82 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
82citing 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

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

  1. Pooled it
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  4. Review
  5. Safety considerations of gene-based therapies for Alzheimer's disease.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2026
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22 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

2 authors.

Jichao SunDepartment of Geriatrics, Shenzhen People's Hospital (The Second Clinical Medical College, Jinan University; The First Affiliated Hospital, Southern University of Science and Technology), Shenzhen, China.
Subhojit RoyDepartment of Pathology, University of California, San Diego, La Jolla, CA, USA. sroy@ucsd.edu.ORCID http://orcid.org/0000-0002-1571-2735

Funding

Slow axonal transport of cytosolic cargoes by dynamic-recruitment - a new trafficR01NS075233 · NINDS · UNIVERSITY OF WISCONSIN-MADISON · PI ROY, SUBHOJIT · 2012 to 2023
$3.4M
Pathophysiologic roles of alpha-synuclein at the synapseR01NS111978 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI ROY, SUBHOJIT · 2019 to 2023
$2.4M
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the BrainUG3NS111688 · NINDS · UNIVERSITY OF WISCONSIN-MADISON · PI EMBORG, MARINA, GONG, SHAOQIN - · 2018 to 2020
$2.3M
Trafficking and Endosomal Sorting of APP and BACE-1R01AG048218 · NIA · UNIVERSITY OF WISCONSIN-MADISON · PI FRIEDL, ANDREAS · 2014 to 2018
$1.6M
A CRISPR-Cas9 screen to identify genetic modifiers of APP/BACE-1 interactionsR21AG052404 · NIA · UNIVERSITY OF WISCONSIN-MADISON · PI FRIEDL, ANDREAS · 2016 to 2017
$408k
NIA NIH HHS R01 AG048218NIA NIH HHS R21 AG052404NINDS NIH HHS R01 NS075233NINDS NIH HHS R01 NS111978NINDS NIH HHS UG3 NS111688
6 · The paper itself

Abstract

Gene therapy is making a comeback. With its twin promise of targeting disease etiology and 'long-term correction', gene-based therapies (defined here as all forms of genome manipulation) are particularly appealing for neurodegenerative diseases, for which conventional pharmacologic approaches have been largely disappointing. The recent success of a viral-vector-based gene therapy in spinal muscular atrophy-promoting survival and motor function with a single intravenous injection-offers a paradigm for such therapeutic intervention and a platform to build on. Although challenges remain, the newfound optimism largely stems from advances in the development of viral vectors that can diffusely deliver genes throughout the CNS, as well as genome-engineering tools that can manipulate disease pathways in ways that were previously impossible. Surely spinal muscular atrophy cannot be the only neurodegenerative disease amenable to gene therapy, and one can imagine a future in which the toolkit of a clinician will include gene-based therapeutics. The goal of this Review is to highlight advances in the development and application of gene-based therapies for neurodegenerative diseases and offer a prospective look into this emerging arena.

Indexed as

Genetic TherapyAnimalsHumansNeurodegenerative Diseases

Identifiers

PMID33526943
PMCPMC8394447

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.