Gene Technologies

Questions and Answers

What is the primary goal of gene therapy?

To treat or prevent disease by correcting underlying genetic problems.

Name one of the methods being studied in gene therapy.

Replacing a mutated gene with a healthy copy.

How does gene transfer therapy function?

It introduces new genetic material into cells to recover the function of faulty proteins.

What does genome editing achieve in gene therapy?

It allows for precise changes to existing DNA in the cell. Signup and view all the answers

Why is gene therapy currently being tested primarily for certain diseases?

Because it is under study to ensure safety and effectiveness in conditions without other cures. Signup and view all the answers

What role does knocking out a gene play in gene therapy?

It inactivates a mutated gene that is not functioning properly. Signup and view all the answers

What is one potential future application of gene therapy?

To treat disorders by inserting a gene into a patient’s cells. Signup and view all the answers

What is meant by 'turning on' a gene in the context of gene therapy?

Activating a gene to help fight a disease. Signup and view all the answers

What are some of the serious health risks associated with early gene therapy studies?

Toxicity, inflammation, and cancer. Signup and view all the answers

How does the CRISPR-Cas9 technique function in gene editing?

CRISPR uses a guide molecule to locate a specific gene, while Cas9 enzyme either changes or cuts the gene. Signup and view all the answers

What is meant by 'correctional' and 'enhancement' in the context of CRISPR-Cas9?

'Correctional' refers to fixing mutated genes, while 'enhancement' involves improving genes for desirable traits. Signup and view all the answers

What ethical concerns arise from germline editing using CRISPR technology?

Concerns include the potential for permanent mutations, misuse of technology, and ethical issues surrounding designer babies. Signup and view all the answers

Discuss a potential social advantage of gene technology mentioned in the content.

Improved crop yields through genetic modifications can enhance nutrition and health in developing countries. Signup and view all the answers

What is a notable con of gene technology in terms of societal impact?

It could increase the gap between social classes by making access to genetic enhancements unequal. Signup and view all the answers

Why is regulating CRISPR technology considered challenging?

The technology is cheap and readily available, making it difficult to manage its use effectively. Signup and view all the answers

Give an example of how gene technology has improved medicine, as discussed.

The insertion of the insulin gene has led to improved treatment for diabetes. Signup and view all the answers

Study Notes

Gene Therapy Overview

Gene therapy treats or prevents diseases by addressing genetic disorders.
Aims to correct genetic problems with the potential to replace drugs or surgery in the future.
Involves experimental techniques targeting various approaches:
- Replacing mutated genes with healthy copies.
- Knocking out malfunctioning mutant genes.
- Introducing new genes to combat diseases.
Currently under study for diseases lacking alternative treatments.

Objectives of Gene Therapy

Correct genetic alterations for proper gene function.
Activate beneficial genes to combat diseases.
Deactivate improperly functioning genes.
Remove detrimental DNA impacting gene functionality.

Mechanisms of Gene Therapy

Genetic code alteration aims to restore functions of critical proteins.
Gene transfer therapy introduces normal gene copies or new instructions to assist cellular function.
Genome editing allows precise modifications in DNA, including addition, removal, or alteration of genetic material.

Safety Concerns in Gene Therapy

Early studies indicated serious risks like toxicity, inflammation, and cancer.
Newer techniques are designed to minimize severe immune reactions and cancer risks.
Unpredictable reactions remain a challenge due to the relative novelty of the field.

CRISPR-Cas9 Technology

A gene editing method that utilizes a guide molecule (Crispr) to target specific DNA regions.
The Cas9 enzyme can modify gene functions or excise genes entirely.
CORRECTIONAL approach focuses on removing defective genes and replacing them.
ENHANCEMENT applies to theoretically enhancing genetic traits.

Gene Technology Considerations

Social Pros:
- Reduces genetic disease risk, enhances understanding of genetics, potentially increases life span.
- Aids in preventing inheritance of genetic traits and diseases.
Social Cons:
- Embryo termination risks, irreversible germline edits, and potential for unintended genome mutations.
- Concerns about misuse of technology and impact on gene pool diversity.

Ethical and Regulatory Issues

Questions surrounding access to genetic enhancements, who controls technology, and the distribution of financial gains.
Potential societal gaps in access to gene editing technologies.
Challenges of regulation due to accessibility and affordability.

Potential Societal Benefits

Crop improvement for nutrition, such as beta-carotene enriched golden rice.
Enhanced medicine through better diagnostics and vaccines, including insulin gene therapy.
Environmental benefits, like pest-resistant crops (BT cotton) reducing pollution in waterways.
Gene technology promises improved life spans and health outcomes.