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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Diarrhea-predominant irritable bowel syndrome (IBS-D) is a subtype of IBS characterized primarily by frequent, loose, or watery stools, abdominal pain, and abdominal discomfort. Therapeutic approaches to managing IBS-D include dietary changes, stress management techniques, and pharmaceutical interventions.
Two specific drugs used in the treatment are alosetron (Lotronex) and eluxadoline (Viberzi). Alosetron, a 5-HT3 antagonist, works by slowing the movement of stools in the gut, reducing bowel...
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Induction of β-Defensin 1 (DEFB1) via synthetic mRNA reduces Cryptosporidium parvum infection in human intestinal cells.

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Related Experiment Video

Updated: Sep 21, 2025

Studying Cryptosporidium Infection in 3D Tissue-derived Human Organoid Culture Systems by Microinjection
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RNA-Based Therapy for Cryptosporidium parvum Infection: Proof-of-Concept Studies.

A Castellanos-Gonzalez1, A Sadiqova1, J Ortega-Mendez1

  • 1Department of Internal Medicine, Infectious Diseases Division, University of Texas Medical Branch, Galveston, Texas, USA.

Infection and Immunity
|June 1, 2022
PubMed
Summary

Novel RNA therapies show promise against Cryptosporidium, a major cause of childhood diarrhea. Single-stranded RNA/Argonaute complexes effectively reduced parasite levels in lab and animal models, offering a potential new treatment for cryptosporidiosis.

Keywords:
ArgonauteCryptosporidiumNDKcryptosporidiosisgene silencingsiRNA

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Area of Science:

  • Parasitology
  • Molecular Biology
  • RNA Therapeutics

Background:

  • Cryptosporidium infection causes severe diarrhea in children, leading to significant mortality.
  • Current treatment, nitazoxanide, has limited efficacy, especially in vulnerable populations.
  • There is an urgent need for alternative therapies against Cryptosporidium.

Purpose of the Study:

  • To evaluate the anticryptosporidial activity of single-stranded RNA (ssRNA)/Argonaute (Ago) complexes.
  • To investigate the silencing of Cryptosporidium parvum nucleoside diphosphate kinase (NDK) using this novel RNA interference approach.
  • To establish proof-of-concept for ssRNA/Ago complexes as a potential treatment for cryptosporidiosis.

Main Methods:

  • Development of ssRNA/Ago complexes targeting essential genes in Cryptosporidium.
  • In vitro studies using infected cell cultures to assess parasite burden reduction.
  • In vivo studies in mouse models, including oral delivery of lipid nanoparticle-encapsulated complexes and assessment of oocyst shedding.

Main Results:

  • A 3-day treatment with anti-sense NDK ssRNA/Ago complexes reduced parasite burden by approximately 98% in infected cells.
  • ssRNA/Ago complexes encapsulated in lipid nanoparticles were successfully delivered to intestinal epithelial cells in mice.
  • Treatment with NDK ssRNA/Ago complexes significantly reduced oocyst shedding in a cryptosporidiosis-mouse model.

Conclusions:

  • Antisense RNA-based therapy using ssRNA/Ago complexes demonstrates significant anticryptosporidial activity.
  • This approach shows potential for effective delivery to intestinal cells and reduction of parasite shedding in vivo.
  • ssRNA/Ago complexes represent a promising alternative therapeutic strategy for cryptosporidiosis treatment.