Explained: What is gene therapy? How is DNA used to treat genetic disorders?

TOI Education / Oct 26, 2023, 16:35 IST

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Explore the revolutionary field of gene therapy, where the power of DNA is harnessed to treat genetic disorders and potentially cure previously incurable diseases.

Explained: What is gene therapy? How is DNA used to treat genetic disorders?

<p>Gene therapy (representative image)<br></p>

Gene therapy is a cutting-edge medical field that holds the promise of treating genetic disorders by altering the very building blocks of life: DNA. In this explainer, we'll delve into the fascinating world of gene therapy, uncovering how it works and the potential it holds for transforming medicine.
Understanding Gene Therapy
At its core, gene therapy is a medical approach that involves modifying or manipulating a person's genes to treat or prevent disease.It's a groundbreaking departure from traditional treatments that often focus on managing symptoms rather than addressing the root cause.
The Role of DNA

DNA, the famous double helix, is the genetic blueprint that contains the instructions for building and operating every part of your body. In gene therapy, scientists and medical professionals target specific genes within your DNA to correct or replace faulty genetic instructions.
Types of Gene Therapy
Gene therapy comes in various forms, but two primary approaches dominate the field:

Somatic Gene Therapy: This approach targets non-reproductive cells in the body (most cells). It aims to treat diseases in the individual patient and is not passed on to future generations.
Germline Gene Therapy: This technique focuses on altering genes in reproductive cells (sperm or egg cells) and is intended to be inherited by offspring. It's a much more complex and controversial area due to ethical and safety concerns.

The Process of Gene Therapy

Gene therapy typically follows these general steps:

Identification: Scientists identify the specific gene responsible for a genetic disorder.
Correction: Using various methods, they introduce a healthy copy of the gene or repair the faulty one.
Delivery: The corrected gene is delivered into the patient's body, often using modified viruses as carriers or other delivery methods.
Integration: The corrected gene integrates into the patient's DNA and starts producing the missing or functioning protein.
Monitoring: Continuous monitoring helps assess the therapy's effectiveness and safety.

Applications of Gene Therapy
Gene therapy has shown promise in treating a wide range of genetic disorders, including:

Cystic Fibrosis
Hemophilia
Muscular Dystrophy
Sickle Cell Anemia
Certain Types of Cancer
Blindness caused by genetic mutations

Challenges and Ethical Considerations
While gene therapy offers immense potential, it also raises significant challenges. These include concerns about safety, off-target effects, and the long-term consequences of genetic modifications. Ethical questions about germline gene editing, accessibility, and affordability are also hotly debated.
The Future of Gene Therapy
As technology advances and our understanding of genetics deepens, the future of gene therapy holds promise for even more transformative treatments. It's an exciting frontier where science fiction becomes science fact, offering hope to patients and their families affected by genetic disorders.
Conclusion: A Revolution in Medicine
Gene therapy represents a remarkable advancement in medical science, where the manipulation of DNA can potentially cure diseases once thought incurable. While challenges and ethical considerations remain, the potential to improve lives and change the course of medicine is undeniable.
Gene therapy exemplifies the power of human innovation and determination to conquer the most complex challenges in the pursuit of a healthier, disease-free future.
(This content is generated with the assistance of Artificial Intelligence)

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