Evidence map›Paper›PMID 32623324›Full record

ReviewCurrent opinion in genetics & development2020

Nucleic acid therapeutics in neurodevelopmental disease.

Audrey M Winkelsas, Kenneth H Fischbeck

Open access · hybridAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Current opinion in genetics & development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
0.5field-weighted citation impact, top 37% 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

6 citing papers in PubMed, 11 citations in OpenAlex.

  1. Review
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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 at 2 institutions in 2 countries.

Audrey M WinkelsasNeurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, United States; Nuffield Department of Medicine, University of Oxford, Oxford, United Kingdom.
Kenneth H FischbeckNeurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, United States. Electronic address: kf@ninds.nih.gov.
National Institutes of Health · USUniversity of Oxford · GB

Funding

Studies of Hereditary Neurological Disease: Disease MechanismsZIANS003038 · NINDS · NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE · PI FISCHBECK, KENNETH · 2009 to 2023
$19.5M
Studies Of Hereditary Neurological Disease: Clinical TrialsZ01NS003037 · NINDS · NEUROLOGICAL DISORDERS AND STROKE · PI FISCHBECK, KENNETH · 2007 to 2008
$2.6M
Intramural NIH HHS Z01 NS003037
6 · The paper itself

Abstract

Nucleic acid therapeutics allow sequence-based targeting of mutation-harboring genes. They can be used to increase the expression and function of disease genes or to decrease the expression of toxic gene products. Antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and gene-replacement therapies have received FDA approval, and in vivo gene editing applications are currently under development. Special consideration should be given to target engagement in neurons and amelioration of neurological phenotypes. Here we discuss the uses and limitations of different nucleic acid therapeutics, highlighting examples in the clinical and pre-clinical application of these modalities for the treatment of neurodevelopmental diseases.

Indexed as

Gene EditingGenetic TherapyAnimalsHumansNeurodevelopmental DisordersOligonucleotides, AntisenseRNA, Small InterferingOligonucleotides, AntisenseRNA, Small Interfering

Identifiers

PMID32623324
PMCPMC11441424
OpenAlexW3039102645

What OpenQuestion holds

Textmetadata
LicenceTDM
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.