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Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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Therapeutic RNA Delivery for COVID and Other Diseases

Curtis Dobrowolski1, Kalina Paunovska1, Marine Z C Hatit1

  • 1Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University School of Medicine, Atlanta, GA, 30332, USA.

Advanced Healthcare Materials
|March 4, 2021
PubMed

Insights

RNA drug delivery faces biological hurdles, but advancements in small interfering RNA (siRNA) for liver disease and messenger RNA (mRNA) for COVID-19 vaccines show promise. New methods are improving delivery to diverse tissues.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Drug Delivery Systems

Background:

  • RNA therapeutics offer potential for treating diseases by modulating gene expression and protein production.
  • Current RNA-based therapies are limited by challenges in delivering therapeutic quantities of RNA to target cells in vivo.
  • Successful in vivo delivery is critical for the widespread development and application of RNA drugs.

Purpose of the Study:

  • To review biological challenges in in vivo RNA drug delivery.
  • To discuss successful RNA drug delivery strategies, including siRNA for liver disease and mRNA for COVID-19 vaccination.
  • To highlight novel research aimed at improving RNA delivery to new tissues.

Main Methods:

  • Review of recent studies on biological barriers to in vivo RNA delivery.
  • Analysis of clinical applications of RNA drugs, specifically siRNA and mRNA.
  • Description of high-throughput in vivo nanoparticle DNA barcoding assays for delivery testing.

Main Results:

  • Significant progress has been made in overcoming biological hurdles for RNA drug delivery.
  • siRNA has shown efficacy in treating liver diseases, and mRNA vaccines have been successful against COVID-19.
  • Novel nanoparticle delivery systems and high-throughput assays are being developed to expand RNA therapy applications.

Conclusions:

  • Despite delivery challenges, RNA-based therapies are demonstrating clinical success.
  • Continued research into delivery mechanisms and nanoparticle technologies is crucial for advancing RNA therapeutics.
  • Improved in vivo delivery will broaden the scope of diseases treatable with RNA-based drugs.
Keywords:
COVIDLNPRNA therapyin vivo deliveryvaccine

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