Gene therapy is a therapeutic approach that involves the introduction, alteration, or removal of genetic material within an individual’s cells to treat or prevent a specific disease or medical condition. It aims to address genetic disorders caused by mutations or abnormalities in the DNA sequence.
The primary goal of gene therapy is to correct or compensate for the underlying genetic defect by introducing functional genes or modifying existing genes. This can be achieved through various techniques, including the delivery of therapeutic genes using viral vectors, direct injection of naked DNA or modified RNA, or genome editing tools such as CRISPR-Cas9.
Gene therapy can target both somatic cells and germ cells. Somatic cell gene therapy targets non-reproductive cells, aiming to treat the individual patient. In contrast, germ line gene therapy targets reproductive cells, with the goal of introducing heritable changes that can be passed on to future generations.
The implementation of gene therapy requires a thorough understanding of the disease’s genetic basis, the specific genes involved, and the appropriate delivery system to reach the target cells or tissues. It also involves careful consideration of ethical, safety, and regulatory aspects, including informed consent, potential off-target effects, and long-term monitoring of the treatment’s efficacy and safety.
Gene therapy holds promise for the treatment of a wide range of diseases, including genetic disorders, cancer, cardiovascular diseases, neurodegenerative disorders, and infectious diseases. While still an evolving field, gene therapy has shown notable success in certain conditions, providing potential long-term solutions and improving the quality of life for affected individuals. Ongoing research and technological advancements continue to expand the possibilities and refine the efficacy and safety of gene therapy approaches.
Read Also: What Is Gene Therapy? – Definition, Types, Vectors, Applications
Gene therapy is a technique that involves:
a) Modifying DNA sequences in bacteria
b) Treating genetic diseases by altering genes
c) Enhancing protein production in cells
d) Promoting gene mutation in humans
Answer: b) Treating genetic diseases by altering genes
Which of the following is a goal of gene therapy?
a) Repairing damaged genes
b) Replacing faulty genes
c) Introducing therapeutic genes
d) All of the above
Answer: d) All of the above
The primary aim of gene therapy is to:
a) Cure genetic diseases
b) Prevent genetic disorders
c) Enhance athletic performance
d) Promote genetic diversity
Answer: a) Cure genetic diseases
In gene therapy, therapeutic genes are introduced into cells using:
a) Viral vectors
b) Bacterial vectors
c) Naked DNA
d) All of the above
Answer: d) All of the above
Which type of viral vector is commonly used in gene therapy?
a) Retrovirus
b) Adenovirus
c) Lentivirus
d) All of the above
Answer: d) All of the above
The purpose of using viral vectors in gene therapy is to:
a) Deliver therapeutic genes into target cells
b) Promote viral replication
c) Generate mutations in the host genome
d) Induce immune responses
Answer: a) Deliver therapeutic genes into target cells
Ex vivo gene therapy involves:
a) Modifying genes in living organisms
b) Directly administering gene therapies to patients
c) Removing cells from the patient’s body, modifying them, and reintroducing them
d) None of the above
Answer: c) Removing cells from the patient’s body, modifying them, and reintroducing them
In vivo gene therapy involves:
a) Modifying genes in living organisms
b) Administering gene therapies directly to patients
c) Removing cells from the patient’s body, modifying them, and reintroducing them
d) None of the above
Answer: b) Administering gene therapies directly to patients
The first successful gene therapy trial was conducted for the treatment of:
a) Cystic fibrosis
b) Hemophilia
c) Severe combined immunodeficiency (SCID)
d) Parkinson’s disease
Answer: c) Severe combined immunodeficiency (SCID)
Gene therapy can be used to target diseases that are caused by:
a) Single gene mutations
b) Environmental factors
c) Bacterial infections
d) All of the above
Answer: a) Single gene mutations
Which of the following diseases has been successfully treated using gene therapy?
a) Leukemia
b) Blindness
c) Muscular dystrophy
d) All of the above
Answer: d) All of the above
Gene therapy can be administered to target cells or tissues in the body, including:
a) Blood cells
b) Liver cells
c) Lung cells
d) All of the above
Answer: d) All of the above
Gene therapy has the potential to provide:
a) Permanent treatment for genetic diseases
b) Temporary relief from symptoms
c) Prevention of genetic disorders
d) None of the above
Answer: a) Permanent treatment for genetic diseases
One of the challenges in gene therapy is:
a) Targeting specific cells or tissues
b) Ensuring long-term gene expression
c) Minimizing immune responses
d) All of the above
Answer: d) All of the above
Gene editing techniques, such as CRISPR-Cas9, can be used in gene therapy to:
a) Correct gene mutations
b) Delete specific genes
c) Insert new genes
d) All of the above
Answer: d) All of the above
Gene therapy is being explored as a potential treatment for:
a) Cancer
b) Neurodegenerative diseases
c) Cardiovascular diseases
d) All of the above
Answer: d) All of the above
The field of gene therapy has evolved significantly in recent years due to advancements in:
a) Genetic engineering techniques
b) Gene delivery systems
c) Understanding of disease mechanisms
d) All of the above
Answer: d) All of the above
Gene therapy carries certain risks, including:
a) Immune reactions
b) Off-target effects
c) Insertional mutagenesis
d) All of the above
Answer: d) All of the above
Ethical considerations in gene therapy include:
a) Informed consent
b) Access to treatment
c) Germline gene editing
d) All of the above
Answer: d) All of the above
The future of gene therapy holds potential for:
a) Personalized medicine
b) Targeted treatments
c) Cure for previously incurable diseases
d) All of the above
Answer: d) All of the above
