ReviewCommunications biology2024
Between hope and reality: treatment of genetic diseases through nucleic acid-based drugs.
Review in Communications biology, 2024. 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 20 papers.
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.
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.
Who cites it
20 citing papers in PubMed.
- Nanocarrier-Based Gene Delivery Systems: Mechanisms, Clinical Translation, and Future Perspectives.Biotechnology and bioengineering · 2026Review
- Review
- TIA1 depletion enhances CLSTN1 exon 11 inclusion to facilitate Epithelial-to-Mesenchymal transition and breast cancer metastasis.Oncogene · 2026Article
- Functional characterization and allele-specific RNA interference-based rescue of KCNH2 p.F68C variant associated with long QT syndrome.Molecular genetics and genomics : MGG · 2026Article
- CRISPR-Cas9-mediated therapeutics: Current clinical trials and therapy approval landscape to treat human diseases.Molecular therapy. Nucleic acids · 2026Review
- Toward Safe and Effective Gene Therapy: Non-Viral Nanostructured Delivery Systems.International journal of nanomedicine · 2026Review
- TheInternational journal of molecular sciences · 2025Review
- The current and future landscape of RNA-based therapies and diagnostics.Communications medicine · 2025Article
- Translational Advances in Lipid Nanoparticle Drug Delivery Systems for Cancer Therapy: Current Status and Future Horizons.Pharmaceutics · 2025Review
- NGLY1 as an Emerging Critical Modulator for Neurodevelopment and Pathogenesis in the Brain.International journal of molecular sciences · 2025Review
- Huntington's chorea: emerging fields in therapeutics (Review).Neurogenetics · 2025Review
- Decrypting the Immune Symphony for RNA Vaccines.Vaccines · 2025Review
- Transfection Technologies for Next-Generation Therapies.Journal of clinical medicine · 2025Review
- Advances in locally administered nucleic acid therapeutics.Bioactive materials · 2025Review
- Advances in Duchenne Muscular Dystrophy: Diagnostic Techniques and Dystrophin Domain Insights.International journal of molecular sciences · 2025Review
- Lymphoblastoid andFrontiers in bioengineering and biotechnology · 2025Article
- A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases.Nature communications · 2024Review
- Construction of aMolecular therapy. Nucleic acids · 2024Article
- Emerging Strategies in Drug Development and Clinical Care in the Era of Personalized and Precision Medicine.Pharmaceutics · 2024Article
- The Roadmap toward Personalized Medicine: Challenges and Opportunities.Journal of personalized medicine · 2024Article
Corrections and comments
- Erratum issued
Authors and funding
5 authors.
Funding
Abstract
Rare diseases (RD) affect a small number of people compared to the general population and are mostly genetic in origin. The first clinical signs often appear at birth or in childhood, and patients endure high levels of pain and progressive loss of autonomy frequently associated with short life expectancy. Until recently, the low prevalence of RD and the gatekeeping delay in their diagnosis have long hampered research. The era of nucleic acid (NA)-based therapies has revolutionized the landscape of RD treatment and new hopes arise with the perspectives of disease-modifying drugs development as some NA-based therapies are now entering the clinical stage. Herein, we review NA-based drugs that were approved and are currently under investigation for the treatment of RD. We also discuss the recent structural improvements of NA-based therapeutics and delivery system, which overcome the main limitations in their market expansion and the current approaches that are developed to address the endosomal escape issue. We finally open the discussion on the ethical and societal issues that raise this new technology in terms of regulatory approval and sustainability of production.
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What OpenQuestion holds
Registered trials
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.