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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...

Rous Sarcoma Virus (RSV) and Cancer

Rous Sarcoma Virus (RSV) and Cancer

Rous Sarcoma virus or RSV was discovered by F. Peyton Rous in the year 1911 as a filterable transmissible agent that could cause tumors in chickens. He won a Nobel Prize for this discovery in 1966. His experiments clearly demonstrated that some cancers could be caused by infectious agents and led to the discovery of many more cancer-causing viruses in animals as well as humans.
RSV is a retrovirus that contains two copies of a plus-strand RNA genome. Its genome consists of four main open...

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Related Experiment Video

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Quantification of Adeno-Associated Viral Genomes in Purified Vector Samples by Digital Droplet Polymerase Chain Reaction

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Published on: October 11, 2024

HSV Amplicons in Gene Therapy.

N Frenkel¹, R Sarid

¹Department of Cell Research and Immunology, Tel Aviv University, Ramat Aviv, Tel Aviv, Israel.

Methods in Molecular Medicine

|March 5, 2011

Summary

Herpes simplex virus (HSV) amplicons serve as effective gene therapy vectors, delivering foreign genes into various cell types, including non-dividing neurons. This offers a promising alternative to current retrovirus vectors for neurological applications.

Area of Science:

Molecular Biology
Virology
Gene Therapy

Background:

Herpes simplex virus (HSV) amplicons are defective viral vectors.
These vectors can introduce amplified foreign genes into eukaryotic cells.
Existing retrovirus vectors require cell division, limiting their use in non-dividing cells like neurons.

Purpose of the Study:

To evaluate HSV amplicons as potential gene therapy vectors for targeting both dividing and non-dividing cells.
To highlight the advantage of HSV amplicons over retrovirus vectors for neuronal gene delivery.

Main Methods:

Utilizing defective Herpes simplex virus (HSV) amplicon vectors.
Introducing amplified foreign genes into various eukaryotic cell types.
Testing infectivity in different organisms, including rodents, monkeys, and humans.

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Main Results:

HSV amplicons successfully introduced foreign genes into diverse cell types (fibroblasts, macrophages, glia, neurons).
These defective viruses infect both mitotic and postmitotic cells.
Demonstrated potential for gene delivery into non-dividing cells, specifically nerve cells.

Conclusions:

Herpes simplex virus (HSV) amplicons are versatile and effective gene therapy vectors.
Their ability to infect non-dividing cells makes them suitable for targeting neurons, overcoming limitations of retrovirus vectors.