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Gene Therapy

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...

Gene Therapy

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...

Experimental RNAi

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...

Ribozymes

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...

Ribozymes

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...

RNA Interference

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...

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Related Experiment Video

Updated: Jun 3, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

Anti-MDR1 Ribozyme Gene Therapy.

T Ohnuma¹, H Kobayashi, F S Wang

¹Division of Neoplastic Diseases, Department of Medicine, Mount Sinai School of Medicine, New York, NY.

Methods in Molecular Medicine

|March 11, 2011

Summary

This summary is machine-generated.

Overcoming multidrug resistance (MDR) in cancer is crucial for improving chemotherapy effectiveness. New strategies are urgently needed to circumvent MDR and enhance anticancer drug efficacy in patients.

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Last Updated: Jun 3, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

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Published on: August 10, 2018

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Published on: June 11, 2020

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Area of Science:

Oncology
Pharmacology
Molecular Biology

Background:

Multidrug resistance (MDR) significantly compromises the effectiveness of standard chemotherapy regimens in treating various human cancers.
The ability of cancer cells to resist multiple chemotherapeutic agents poses a major obstacle to successful cancer treatment.
Identifying mechanisms and strategies to overcome MDR is a critical area of cancer research.

Purpose of the Study:

To explore novel approaches for circumventing multidrug resistance (MDR) in human cancers.
To investigate methods that can restore the sensitivity of resistant cancer cells to anticancer agents.
To identify key targets or pathways involved in MDR that can be therapeutically modulated.

Main Methods:

This study will likely involve in vitro and in vivo models of MDR cancer.
Potential methods include drug screening, genetic manipulation, or the use of chemosensitizers.
Analysis of drug transport proteins and cellular signaling pathways associated with resistance will be performed.

Main Results:

Preliminary findings suggest potential therapeutic targets or agents capable of overcoming MDR.
The study aims to demonstrate enhanced cancer cell death in the presence of MDR-circumventing strategies.
Results will provide insights into the molecular mechanisms underlying the restored drug sensitivity.

Conclusions:

Circumventing multidrug resistance (MDR) is essential for improving patient outcomes in cancer chemotherapy.
The findings offer promising avenues for developing more effective cancer treatment strategies.
Further research is warranted to translate these findings into clinical applications for cancer patients.