Evidence map›Paper›PMID 36792602›Full record

ReviewTranslational psychiatry2023

Therapeutic strategies for autism: targeting three levels of the central dogma of molecular biology.

Derek Hong, Lilia M Iakoucheva

Erratum issuedOpen access · goldAbstract readReview
In one paragraph

Review in Translational psychiatry, 2023. 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 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed
4.3field-weighted citation impact, top 5% 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

19 citing papers in PubMed, 28 citations in OpenAlex.

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  13. Simultaneous inference for generalized linear models with unmeasured confounders.Journal of the American Statistical Association · 2025
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

2 authors at 1 institution in 1 country.

Derek HongDepartment of Psychiatry, University of California San Diego, La Jolla, CA, USA.ORCID 0000-0002-5921-8798
Lilia M IakouchevaDepartment of Psychiatry, University of California San Diego, La Jolla, CA, USA. lilyak@ucsd.edu.ORCID 0000-0002-4542-5219
University of California San Diego · US

Funding

Integrative functional genomic study of pathways impacted by recurrent autism CNVR01MH108528 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI IAKOUCHEVA, LILIA M, MUOTRI, ALYSSON R. · 2016 to 2020
$2.6M
Evaluating the effect of splicing mutations on isoform networks in autismR01MH109885 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI IAKOUCHEVA, LILIA M, MUOTRI, ALYSSON R. · 2016 to 2020
$2.5M
Cortical organoid models to study autism-associated 16p.11.2.CNVR56MH128365 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI IAKOUCHEVA, LILIA M, MUOTRI, ALYSSON R. · 2022 to 2022
$706k
Rescue of Cul3 haploinsufficiency phenotypes with CRISPR-mediated Cul3 activationR21MH128827 · NIMH · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI IAKOUCHEVA, LILIA M · 2022 to 2023
$435k
NIMH NIH HHS R01 MH108528NIMH NIH HHS R01 MH109885NIMH NIH HHS R21 MH128827NIMH NIH HHS R56 MH128365
6 · The paper itself

Abstract

The past decade has yielded much success in the identification of risk genes for Autism Spectrum Disorder (ASD), with many studies implicating loss-of-function (LoF) mutations within these genes. Despite this, no significant clinical advances have been made so far in the development of therapeutics for ASD. Given the role of LoF mutations in ASD etiology, many of the therapeutics in development are designed to rescue the haploinsufficient effect of genes at the transcriptional, translational, and protein levels. This review will discuss the various therapeutic techniques being developed from each level of the central dogma with examples including: CRISPR activation (CRISPRa) and gene replacement at the DNA level, antisense oligonucleotides (ASOs) at the mRNA level, and small-molecule drugs at the protein level, followed by a review of current delivery methods for these therapeutics. Since central nervous system (CNS) penetrance is of utmost importance for ASD therapeutics, it is especially necessary to evaluate delivery methods that have higher efficiency in crossing the blood-brain barrier (BBB).

Indexed as

Autism Spectrum DisorderAutistic DisorderBlood-Brain BarrierDrug Delivery SystemsHumansMolecular Biology

Identifiers

PMID36792602
PMCPMC9931756
OpenAlexW4321002254

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.