ReviewMolecular therapy : the journal of the American Society of Gene Therapy2021
Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 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
30 citing papers in PubMed, 52 citations in OpenAlex.
- Small Interfering RNA (siRNA) for Cardiorenal Disease: Mechanistic Insights from Preclinical and Clinical Studies.Journal of cardiovascular translational research · 2025Review
- Modeling and Simulation Identifies Endocytosis Uptake Rate and Fraction Unbound as Important Predictors of Oligonucleotide Pharmacokinetics.CPT: pharmacometrics & systems pharmacology · 2025Article
- Strategies and efforts in circumventing the emergence of antiviral resistance against conventional antivirals.npj antimicrobials and resistance · 2025Review
- Highly efficient nucleic acid encapsulation method for targeted gene therapy using antibody conjugation system.Molecular therapy. Nucleic acids · 2024Article
- Nucleic acid drugs: recent progress and future perspectives.Signal transduction and targeted therapy · 2024Review
- The RNA receptor RIG-I binding synthetic oligodeoxynucleotide promotes pneumonia survival.JCI insight · 2024Article
- Current status and trends in small nucleic acid drug development: Leading the future.Acta pharmaceutica Sinica. B · 2024Review
- Potential Use of MicroRNA Technology in Thalassemia Therapy.Journal of clinical medicine research · 2024Review
- Population pharmacokinetics of imetelstat, a first-in-class oligonucleotide telomerase inhibitor.CPT: pharmacometrics & systems pharmacology · 2024Article
- A comparative analysis of peptide-delivered antisense antibiotics using diverse nucleotide mimics.RNA (New York, N.Y.) · 2024Article
- An engineered poly(A) tail attenuates gut ischemia/reperfusion-induced acute lung injury.Surgery · 2024Article
- Review
- Exosomal long noncoding RNA MLETA1 promotes tumor progression and metastasis by regulating the miR-186-5p/EGFR and miR-497-5p/IGF1R axes in non-small cell lung cancer.Journal of experimental & clinical cancer research : CR · 2023Article
- Updated Considerations for the Immunopharmacological Aspects of the "Talented mRNA Vaccines".Vaccines · 2023Review
- An Overview of the Stability and Delivery Challenges of Commercial Nucleic Acid Therapeutics.Pharmaceutics · 2023Review
- Recent progress of aptamer‒drug conjugates in cancer therapy.Acta pharmaceutica Sinica. B · 2023Review
- Hypoxia-induced lncRNA MRVI1-AS1 accelerates hepatocellular carcinoma progression by recruiting RNA-binding protein CELF2 to stabilize SKA1 mRNA.World journal of surgical oncology · 2023Article
- Identification of a novel small-molecule inhibitor of miR-29b attenuates muscle atrophy.Molecular therapy. Nucleic acids · 2023Article
- Allometric-like scaling of AAV gene therapy for systemic protein delivery.Molecular therapy. Methods & clinical development · 2022Article
- Targeting oncogenic KRAS with molecular brush-conjugated antisense oligonucleotides.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Oligonucleotide therapeutics are a novel promising class of drugs designed to specifically target either coding or non-coding RNA molecules to revolutionize treatment of various diseases. During preclinical development, investigations of the pharmacokinetic characteristics of these oligonucleotide-based drug candidates are essential. Oligonucleotides possess a long history of chemical modifications to enhance their stability and binding affinity, as well as reducing toxicity. Phosphorothioate backbone modifications of oligonucleotides were a hallmark of this development process that greatly enhanced plasma stability and protein binding of these agents. Modifications such as 2'-O-methylation further improved stability, while other modifications of the ribose, such as locked nucleic acid (LNA) modification, significantly increased binding affinity, potency, and tissue half-life. These attributes render oligonucleotide therapeutics able to regulate protein expression in both directions depending on the target RNA. Thus, a growing interest has emerged using these oligonucleotides in the treatment of neurodegenerative and cardiac disorders as well as cancer, since the deregulation of certain coding and non-coding RNAs plays a key role in the development of these diseases. Cutting edge research is being performed in the field of non-coding RNAs, identifying potential therapeutic targets, and developing novel oligonucleotide-based agents that outperform classical drugs. Some of these agents are either in clinical trials showing promising results or are already US Food and Drug Administration (FDA) approved, with more oligonucleotides being developed for therapeutic purposes. This is the advent of mechanism-based next-generation therapeutics for a wide range of diseases.
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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.