Conquering Diseases – Gene Therapy
- Vaibhavi Godkhindi
- Sep 6, 2023
- 2 min read
Human beings have a comprehensive understanding of various diseases, some caused by bacteria, viruses, or other microorganisms, while others have genetic origins—either acquired, like cancer, or inherited, such as hemophilia.
While a wide range of antibiotics, pharmaceuticals, and therapeutic agents exist for microorganism-related diseases, options for genetic diseases are significantly limited. Within the human cell, approximately 25,000 genes exist, and even a minor alteration or mutation can lead to disease or physical instability.
Gene therapy presents a sophisticated technique for addressing these mutated or faulty genes, utilizing DNA as a therapeutic agent. It can replace nonfunctional or aberrant genes with adequately functioning ones and deactivate mutated genes responsible for diseases or activate protective genes. In some cases, the immune system struggles to identify these defects, requiring gene therapy to bolster the immune response.
In 1990, a groundbreaking moment occurred when Dr. W. French, Kenneth Culver, and Michael Blaese administered gene therapy to Ashanti De Sila, a patient with ADA-SCID.
There are two fundamental types of gene therapy: somatic gene therapy and germline gene therapy.
Somatic cell gene therapy treats diseases stemming from gene defects in somatic cells. In these cases, altering a patient's genes for therapeutic purposes doesn't affect the likelihood of passing the disease to offspring, making it the most common form of gene therapy.
On the other hand, germline gene therapy focuses on modifying germ cell genes, enabling inherited alterations by future generations. This approach introduces genetic changes in early-stage embryos, which integrate into all body cells. Currently, germline gene therapy is not being explored in larger animals or humans due to safety concerns.
Inserting genes into cells is a complex process. Vectors, primarily viruses, serve as agents for gene delivery into cells. These viral vectors are prone to destabilization, potentially causing disease. Moreover, the immune system may recognize these viruses as foreign, triggering an immune response. Precision ensures accurate gene insertion, preventing unintended mutations or instability.
The long-term implications of gene manipulation remain incompletely understood. Ethical debates persist regarding the creation of "ideal" human beings. Gene therapy, a relatively uncharted field, relies heavily on theories that may entail risks for human subjects. In many cases, theories can diverge from their intended outcomes, carrying profound implications.
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