CSIR NET Life Science Exam > CSIR NET Life Science Videos > Crash Course for CSIR NET Life Sciences (English) > Human Gene Therapy, Types of Gene Therapy - 4
Human Gene Therapy, Types of Gene Therapy - 4 Video Lecture | Crash Course for CSIR NET Life Sciences (English)
FAQs on Human Gene Therapy, Types of Gene Therapy - 4 Video Lecture - Crash Course for CSIR NET Life Sciences (English)
| 1. What is gene therapy and how does it work? |  |
Ans.Gene therapy is a medical technique that aims to treat or prevent diseases by modifying the genes within an individual's cells. It works by introducing, removing, or altering genetic material within a person's cells to correct or replace defective genes that are responsible for disease development. This can involve delivering healthy copies of genes, repairing faulty genes, or using techniques like CRISPR to edit gene sequences, allowing for potential cures for genetic disorders, some cancers, and viral infections.
| 2. What are the main types of gene therapy? | |
Ans.The main types of gene therapy include somatic gene therapy, germline gene therapy, and ex vivo and in vivo gene therapy. Somatic gene therapy involves altering genes in somatic (non-reproductive) cells and affects only the individual receiving the treatment. Germline gene therapy involves changes to reproductive cells, which can be passed on to future generations. Ex vivo gene therapy involves modifying cells outside the body and then reintroducing them, while in vivo gene therapy delivers therapeutic genes directly into the patient's body.
| 3. What are the potential benefits of gene therapy? | |
Ans.Gene therapy holds several potential benefits, including the ability to treat or even cure genetic disorders that currently have no effective treatments. It can provide long-term solutions by addressing the root cause of diseases rather than just alleviating symptoms. Additionally, gene therapy can enhance the body’s ability to fight diseases, such as certain cancers or viral infections, and may lead to advancements in personalized medicine, where treatments are tailored to an individual's specific genetic makeup.
| 4. What are the risks and challenges associated with gene therapy? | |
Ans.Gene therapy, while promising, comes with several risks and challenges. These can include immune reactions to the introduced genes or vectors, potential insertional mutagenesis where the new genes disrupt existing genes, and the possibility of incomplete or ineffective gene delivery. Ethical concerns also arise, particularly with germline gene therapy, as it involves altering human heredity. Furthermore, the high cost of gene therapy treatments and the need for extensive clinical trials to ensure safety and efficacy present significant hurdles to widespread implementation.
| 5. How has gene therapy evolved over the years? | |
Ans.Gene therapy has evolved significantly since its inception in the late 20th century. Initial attempts in the 1990s faced numerous challenges, including safety issues and regulatory hurdles. However, advancements in genetic engineering technologies, such as the development of viral vectors and CRISPR gene editing, have improved the effectiveness and safety of gene therapies. Recent successes in treating conditions like spinal muscular atrophy and certain inherited retinal diseases have demonstrated the potential of gene therapy, leading to increased investment and research in this field.
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Oct 20, 2025
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