Evidence map›Paper›PMID 38570694›Full record

ReviewNature reviews. Drug discovery2024

RNAi-based drug design: considerations and future directions.

Qi Tang, Anastasia Khvorova

Open access · greenAbstract readReview
In one paragraph

Review in Nature reviews. Drug discovery, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 157 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
157citing papers in PubMed, 1 pooled it
49.3field-weighted citation impact, top 1% 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

157 citing papers in PubMed, 1 synthesis or guideline pooled it, 212 citations in OpenAlex.

  1. Pooled it
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  9. Defect-Engineered BiOAdvanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Article
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  20. Combination siRNA delivery as a therapeutic strategy for ADPKD.bioRxiv : the preprint server for biology · 2026
    Article

97 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 at 1 institution in 1 country.

Qi TangRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.ORCID 0000-0002-8913-0519
Anastasia KhvorovaRNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA. Anastasia.Khvorova@umassmed.edu.ORCID 0000-0001-6928-8071
University of Massachusetts Chan Medical School · US

Funding

Expanding the chemical diversity of therapeutic oligonucleotides to treat neurodegenerative disordersR01NS104022 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ANASTASIA KHVOROVA · 2017 to 2026
$6.0M
Chemical engineering of therapeutic RNAs for extrahepatic deliveryR35GM131839 · NIGMS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ANASTASIA KHVOROVA · 2019 to 2026
$3.1M
Developing a programmable siRNA-based therapeutic platform for gene silencing in the skinK99AR082987 · NIAMS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI TANG, QI · 2023 to 2024
$226k
NIAMS NIH HHS K99 AR082987NIGMS NIH HHS R35 GM131839NINDS NIH HHS R01 NS104022
6 · The paper itself

Abstract

More than 25 years after its discovery, the post-transcriptional gene regulation mechanism termed RNAi is now transforming pharmaceutical development, proved by the recent FDA approval of multiple small interfering RNA (siRNA) drugs that target the liver. Synthetic siRNAs that trigger RNAi have the potential to specifically silence virtually any therapeutic target with unprecedented potency and durability. Bringing this innovative class of medicines to patients, however, has been riddled with substantial challenges, with delivery issues at the forefront. Several classes of siRNA drug are under clinical evaluation, but their utility in treating extrahepatic diseases remains limited, demanding continued innovation. In this Review, we discuss principal considerations and future directions in the design of therapeutic siRNAs, with a particular emphasis on chemistry, the application of informatics, delivery strategies and the importance of careful target selection, which together influence therapeutic success.

Indexed as

Drug DesignRNA InterferenceRNA, Small InterferingAnimalsDrug Delivery SystemsHumansRNA, Small Interfering

Identifiers

PMID38570694
PMCPMC11144061
OpenAlexW4393869156

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.