Non-Viral RNA Delivery During Pregnancy: Opportunities and Challenges.

Small

Department of Chemical Engineering, Carnegie Mellon University, Pittsburgh, PA, 15213, USA.

Published: October 2024

During pregnancy, the risk of maternal and fetal adversities increases due to physiological changes, genetic predispositions, environmental factors, and infections. Unfortunately, treatment options are severely limited because many essential interventions are unsafe, inaccessible, or lacking in sufficient scientific data to support their use. One potential solution to this challenge may lie in emerging RNA therapeutics for gene therapy, protein replacement, maternal vaccination, fetal gene editing, and other prenatal treatment applications. In this review, the current landscape of RNA platforms and non-viral RNA delivery technologies that are under active development for administration during pregnancy is explored. Advancements of pregnancy-specific RNA drugs against SARS-CoV-2, Zika, influenza, preeclampsia, and for in-utero gene editing are discussed. Finally, this study highlights bottlenecks that are impeding translation efforts of RNA therapies, including the lack of accurate cell-based and animal models of human pregnancy and concerns related to toxicity and immunogenicity during pregnancy. Overcoming these challenges will facilitate the rapid development of this new class of pregnancy-safe drugs.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11196389PMC
http://dx.doi.org/10.1002/smll.202306134DOI Listing

Publication Analysis

Top Keywords

non-viral rna
8
rna delivery
8
gene editing
8
pregnancy
5
rna
5
delivery pregnancy
4
pregnancy opportunities
4
opportunities challenges
4
challenges pregnancy
4
pregnancy risk
4

Similar Publications

Double-Stranded RNA-Based Method for Diagnosing Severe Fever with Thrombocytopenia.

J Clin Med

December 2024

Department of Internal Medicine, Division of Infectious Disease, Soonchunhyang University Hospital, Cheonan 31151, Republic of Korea.

: This study explores the potential of using elevated levels of blood double-stranded RNA (dsRNA) as a diagnostic tool for severe fever with thrombocytopenia syndrome (SFTS) infection. : Blood samples from SFTS patients were collected, dsRNA was purified, and total dsRNA expression was quantitatively analyzed using a spiropyran-based method. Comparative analysis was performed using blood samples from healthy individuals and scrub typhus patients with similar symptoms.

View Article and Find Full Text PDF

Endosomal Escape and Nuclear Localization: Critical Barriers for Therapeutic Nucleic Acids.

Molecules

December 2024

Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.

Therapeutic nucleic acids (TNAs) including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA) have emerged as promising treatment strategies for a wide variety of diseases, offering the potential to modulate gene expression with a high degree of specificity. These small, synthetic nucleic acid-like molecules provide unique advantages over traditional pharmacological agents, including the ability to target previously "undruggable" genes. Despite this promise, several biological barriers severely limit their clinical efficacy.

View Article and Find Full Text PDF

, the most common non-viral sexually transmitted parasite, causes more than 270 million infections annually. The infection's outcome varies greatly depending on different factors that include variation in human immune responses, the vaginal microbiome, and the inherent virulence of the strain. Although the pathogenicity of the different strains depends, at least partially, on differential gene expression of virulence genes; the regulatory mechanisms governing this transcriptional control remain incompletely understood.

View Article and Find Full Text PDF

Lipid nanoparticles (LNP) are the most clinically advanced non-viral gene delivery system. While progress has been made for enhancing delivery, cell specific targeting remains a challenge. Targeting moieties such as antibodies can be chemically-conjugated to LNPs however, this approach is complex and has challenges for scaling up.

View Article and Find Full Text PDF
Article Synopsis
  • The Centre of Excellence for the Technologies of Gene and Cell Therapy (CTGCT) has been established at the National Institute of Chemistry in Ljubljana, marking Slovenia’s first center dedicated to precision medicine and cutting-edge therapies.
  • The CTGCT aims to advance cancer immunotherapy and personalized treatments for genetic diseases by developing innovative biomedical tools and collaborating with international institutions for effective therapy development.
  • Its focus on translating research into practice, alongside partnerships with clinicians and patient organizations, positions the CTGCT as a key player in improving access to gene and cell therapies across Slovenia and the broader Eastern European region.
View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!