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RNA therapeutics for disorders of excretory system

Thuy-Duong Vu1, Mai Anh Nguyen1, Adam Jurgoński2

  • 1Center for Biomedicine and Community Health, International School, Vietnam National University, Hanoi, Vietnam.

Progress in Molecular Biology and Translational Science
|February 15, 2024
PubMed

Insights

RNA therapeutics offer novel treatments for excretory system diseases, addressing limitations of current therapies. Small interfering RNA (siRNA) shows significant promise, with one FDA-approved drug and others in clinical trials.

Area of Science:

  • Biomedical Science
  • Molecular Biology
  • Pharmacology

Background:

  • The excretory system is vital for waste removal, but current disease treatments have limitations.
  • Novel therapeutic approaches are needed for excretory system disorders.
  • RNA-based therapeutics have emerged as promising candidates.

Purpose of the Study:

  • To provide an overview of RNA therapeutics for excretory system disorders.
  • To highlight the potential of RNA-based drugs in treating conditions like primary hyperoxaluria type 1, paroxysmal nocturnal hemoglobinuria, urothelial cancer, and renal cancer.

Main Methods:

  • Review of current literature on RNA therapeutics.
  • Analysis of clinical trial data for RNA-based drug candidates.
  • Focus on small interfering RNA (siRNA), antisense oligonucleotides (ASO), and messenger RNA (mRNA) therapeutics.

Main Results:

  • One RNA-based drug, Oxlumo (Lumasiran), is FDA-approved for primary hyperoxaluria type 1.
  • Several other RNA-based candidates are in clinical trials for various excretory system diseases.
  • Small interfering RNA (siRNA) therapeutics are identified as the most promising category.

Conclusions:

  • RNA therapeutics represent a significant advancement in treating excretory system disorders.
  • siRNA, ASO, and mRNA technologies offer new hope for patients with unmet medical needs.
  • Continued research and clinical development are crucial for realizing the full potential of RNA-based treatments.
Keywords:
Disorders of excretory systemDrugsRNA therapeuticsTreatment method

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