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Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Ribozymes02:47

Ribozymes

The term ribozyme is used for RNA that can act as an enzyme. Ribozymes are mainly found in selected viruses, bacteria, plant organelles, and lower eukaryotes. Ribozymes were first discovered in 1982 when Tom Cech’s laboratory observed Group I introns acting as enzymes. This was shortly followed by the discovery of another ribozyme, Ribonulcease P, by Sid Altman’s laboratory. Both Cech and Altman received the Nobel Prize in chemistry in 1989 for their work on ribozymes.
Ribozymes can be...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Ribozymes02:47

Ribozymes

The term ribozyme is used for RNA that can act as an enzyme. Ribozymes are mainly found in selected viruses, bacteria, plant organelles, and lower eukaryotes. Ribozymes were first discovered in 1982 when Tom Cech’s laboratory observed Group I introns acting as enzymes. This was shortly followed by the discovery of another ribozyme, Ribonulcease P, by Sid Altman’s laboratory. Both Cech and Altman received the Nobel Prize in chemistry in 1989 for their work on ribozymes.
Ribozymes can be...
Hepatitis01:25

Hepatitis

Hepatitis is an inflammatory condition of the liver most commonly caused by hepatotropic viruses (A–E), though non-infectious causes such as alcohol and drugs also exist.Hepatitis AHepatitis A virus (HAV) is a non-enveloped RNA virus of the Picornaviridae family. It is primarily transmitted via the fecal-oral route, typically through ingestion of contaminated food or water. After ingestion, HAV enters the bloodstream through the oropharynx or intestinal epithelium and reaches the liver. The...
Viral Hepatitis I: Introduction01:28

Viral Hepatitis I: Introduction

Viral hepatitis is an inflammatory condition of the liver caused by infection with hepatotropic viruses, most commonly hepatitis A, B, C, D, and E. Despite variations in structure and transmission, all viruses mentioned infect hepatocytes and provoke immune responses that can hinder liver function. Additionally, some non-hepatotropic viruses can also lead to hepatic inflammation.Hepatitis A VirusHepatitis A virus (HAV) is transmitted through the fecal–oral route, typically by ingestion of food...

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Related Experiment Video

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High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses
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Ribozyme gene therapy for hepatitis C virus infection

P J Welch1, S Yei, J R Barber

  • 1Immusol Inc., San Diego, CA 92121, USA.

Clinical and Diagnostic Virology
|September 19, 1998
PubMed
Summary

Hepatitis C virus (HCV) gene therapy using ribozymes (Rz) shows promise for developing new antiviral drugs. This approach targets multiple conserved HCV RNA sequences, potentially overcoming drug resistance.

Area of Science:

  • Molecular biology
  • Virology
  • Gene therapy

Background:

  • Hepatitis C virus (HCV) poses a significant challenge due to its high mutation rate, leading to drug resistance in antiviral therapies.
  • Ribozymes (Rz) are RNA molecules with enzymatic activity that can be engineered to target specific RNA sequences.
  • Gene therapy offers a continuous intracellular supply of Rz, potentially preventing the emergence of drug-resistant viral variants.

Purpose of the Study:

  • To investigate the use of viral vectors for delivering ribozyme genes directly into hepatocytes of HCV-infected patients.
  • To degrade the HCV genome and viral mRNAs by targeting multiple conserved RNA sequences simultaneously, thereby circumventing drug resistance.

Main Methods:

  • In vitro transcription of Rzs targeting conserved HCV RNA sequences.

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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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  • In vitro cleavage assays using Rzs to degrade HCV target RNAs.
  • Incorporation of effective Rzs into adeno-associated viral (AAV) and adenoviral (AV) vectors.
  • Testing Rz-expressing viral vectors in tissue culture to inhibit HCV core gene expression.

    • Main Results:

    • Several Rzs demonstrated effective degradation of both positive and negative strands of HCV RNA in vitro.
    • Viral vectors successfully delivered Rz genes into cells, leading to substantial inhibition of HCV gene expression in tissue culture.

    Conclusions:

    • Ribozyme gene therapy presents a viable strategy for developing novel anti-HCV drugs.
    • Initial studies indicate success in using Rz gene therapy to produce antiviral effects against HCV.
    • This approach may offer a solution to the challenge of drug-resistant mutant emergence in HCV therapy.