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Questions and Answers

What type of gene therapy targets non-reproductive cells?

In vivo gene therapy

Germline gene therapy

Somatic gene therapy (correct)

Ex vivo gene therapy

Which of the following is a potential benefit of gene therapy?

Complete elimination of ethical concerns

Guaranteed success in all cases

Immediate recovery from all diseases

Treatment of genetic disorders (correct)

Which form of gene therapy involves modifying cells outside the body?

Germline gene therapy

In vivo gene therapy

Somatic gene therapy

Ex vivo gene therapy (correct)

What is a common concern regarding the safety of gene therapy?

It might lead to unintended genetic mutations. Signup and view all the answers

Which of the following best describes germline gene therapy?

It alters reproductive cells and can affect future generations. Signup and view all the answers

What ethical concern is mainly associated with gene therapy?

Concerns about editing genes for non-medical reasons. Signup and view all the answers

Which statement about the accessibility of gene therapy is true?

The high cost may widen health disparities. Signup and view all the answers

One potential advancement driven by gene therapy is:

The development of new insights and therapies. Signup and view all the answers

What is one of the approaches to gene therapy?

Inactivation of a mutated gene that is functioning improperly Signup and view all the answers

Which of the following best describes the role of viral vectors in gene therapy?

They carry therapeutic genes into cells after being modified. Signup and view all the answers

What is a potential concern regarding long-term effects of gene therapy?

Unforeseen consequences that may emerge after extended periods Signup and view all the answers

What type of gene therapy product uses circular DNA molecules to deliver therapeutic genes?

Plasmid DNA Signup and view all the answers

How are patient-derived cellular gene therapy products created?

Cells are modified outside the patient and reintroduced. Signup and view all the answers

Which statement is true regarding ethical considerations in gene therapy?

Gene therapy should be approached with careful consideration of ethical implications. Signup and view all the answers

What is the primary goal of gene editing?

To disrupt harmful genes or repair mutated genes Signup and view all the answers

Which of the following diseases can be treated with plasmid DNA in gene therapy?

HIV-AIDS Signup and view all the answers

Study Notes

Gene Therapy Overview

Gene therapy is a modern approach to treating hereditary illnesses.
It aims to lessen negative consequences of hereditary conditions.
Requires further research and medical practitioner involvement to realize full potential.
Targets the elimination of currently incurable conditions worldwide.

Lesson Objectives

Describe gene therapy and its types.
Assess potential benefits and drawbacks of gene therapy on global health.

Forms of Gene Therapy

Somatic gene therapy: Targets non-reproductive cells, affecting only the treated individual.
Germline gene therapy: Alters reproductive cells, potentially affecting future generations. Less common due to controversial nature.
Ex vivo gene therapy: Modifies cells outside the body and then reintroduces them.
In vivo gene therapy: Directly targets cells inside the body with therapeutic genes.

Benefits of Gene Therapy

Treatment of genetic disorders: Targets the root cause of inherited diseases like cystic fibrosis, muscular dystrophy, and some cancers.
Personalized medicine: Tailored treatments based on an individual's genetic makeup, increasing efficacy and reducing side effects.
Potential for curing diseases: Addresses the underlying genetic cause, potentially eliminating the disease rather than just managing symptoms.
Advancements in research: Drives innovation in genetic and molecular research, leading to new insights and therapies for various health issues.

Detrimental Effects of Gene Therapy

Safety concerns: Modifying genetic material has potential for unintended effects, off-target mutations, and new health issues, including cancer.
Ethical issues: Gene editing raises concerns around "designer babies" and genetic modifications for non-medical purposes.
Accessibility and inequality: High costs may limit access, exacerbating health disparities between wealthy and low-income populations.
Long-term effects: The long-term consequences of gene therapy may not be fully understood until after extended periods.

Basic Process of Gene Therapy

Replacement: Replacing a defective gene with a healthy copy.
Inactivation: Inactivating a mutated gene that is malfunctioning.
Introduction: Introducing a new gene to help fight a disease.

Variety of Gene Therapy Products

Plasmid DNA: Key component of gene therapy and DNA vaccines, used to treat various diseases including cancer, HIV, Ebola, and malaria. Circular DNA molecules can be engineered to carry therapeutic genes.
Viral vectors: Modified viruses that carry therapeutic genes into cells. Viruses naturally deliver genetic material, and then they are modified to not cause disease, before vectors carry therapeutic genes.
Bacterial vectors: Modified bacteria used to deliver DNA to cells. Genetically modified to not cause infection.
Human gene editing Technology: This technology aims to disrupt harmful or repair mutated genes.

Gene Therapies

Gene therapy modifies a person's genes to treat or prevent diseases.
This corrects defective genes, replacing faulty genes with healthy copies, or introduces new ones to fight diseases.
Inherited genetic disorders (like cystic fibrosis/muscular dystrophy) and acquired diseases (e.g., cancer) can both be targeted..

Gene Therapy Techniques

Gene Replacement: Replacing a defective gene with a functional one.
Gene Editing: Using techniques like CRISPR-Cas9 to directly modify or correct parts of a gene.
Gene Augmentation: Introducing new or modified genes to help the body fight disease or compensate for abnormal genes.
Gene Inhibition: Turning off or blocking genes that cause disease.

Gene Therapy Delivery Methods

In Vivo: Directly into the body.
Ex Vivo: Cells are modified outside the body and then reintroduced.
Gene therapies aim to cure a wide range of diseases by not just reducing symptoms but removing the cause.