Week 14b Evaluation
1.What is gene therapy?
Gene therapy is a medical approach that involves introducing genetic material into a patient’s cells to
treat or prevent disease. It aims to correct genetic disorders by replacing, modifying, or regulating faulty
genes.
2.What are the forms of gene therapy?
a. Gene Replacement Therapy: This involves replacing a faulty gene with a functional one to correct a
genetic disorder.
b. Gene Augmentation Therapy: In this approach, additional copies of a functional gene are introduced
into cells to supplement the deficient gene expression.
c. Gene Editing: Gene editing techniques, such as CRISPR-Cas9, allow precise modification of the DNA
sequence to correct genetic mutations or regulate gene expression.
3.What are the benefits of gene therapy?
a. Treatment of Genetic Disorders: Gene therapy holds promise for treating a wide range of genetic
disorders that were previously considered incurable.
b. Potential Cure for Diseases: It offers the potential to cure diseases at the genetic level, rather than just
managing symptoms.
c. Targeted Treatment: Gene therapy can target specific cells or tissues, minimizing side effects and
improving treatment effectiveness.
d. Personalized Medicine: It enables personalized treatment approaches tailored to an individual’s
genetic makeup.
4.What are the ethical issues on gene therapy?
a. Safety Concerns: There are concerns about the safety of gene therapy, including the risk of unintended
genetic changes or adverse reactions to the treatment.
b. Germline Editing: Editing genes in germline cells (sperm, eggs, or embryos) raises ethical questions
about the potential for heritable genetic modifications and their long-term consequences.
c. Access and Equity: There are ethical considerations regarding the equitable access to gene therapy, as
it may exacerbate existing disparities in healthcare access and affordability.
d. Informed Consent: Ensuring informed consent from patients undergoing gene therapy is crucial,
especially considering the complexity and potential long-term effects of genetic interventions.