What is gene therapy?

Name: Problem 23, Chapter 19: Genetics: A Conceptual Approach
Uploaded: 2020-07-08T03:10:34-08:00
Duration: 1 min 46 s
Channel: Kendrick Buford
Description: What is gene therapy?

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Key Concepts

Vectors for Gene Delivery

Vectors are delivery systems used to transport therapeutic genes into a patient’s cells. Often based on modified viruses, these tools are engineered to carry genetic material safely and efficiently to the targeted cells without causing an adverse reaction.

Somatic vs Germline Gene Therapy

Somatic gene therapy targets non-reproductive cells to treat an individual’s disease without affecting future generations, whereas germline gene therapy involves modifying reproductive cells, which would pass alterations on to offspring. Due to ethical, legal, and safety concerns, current clinical practices predominantly focus on somatic gene therapy.

Gene Therapy

Gene therapy is a biomedical approach that involves modifying a person’s genes to treat or prevent disease. This process typically involves inserting, deleting, or altering genetic material within a patient’s cells to correct diseased gene functions at their source.

Genetic Modification

Genetic modification involves the deliberate alteration of the genetic material of an organism. In the context of gene therapy, it refers to the process of changing gene sequences to restore or introduce normal function, thereby addressing the underlying causes of genetic disorders.

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