Gene Therapy and Its Implications

The video explores the concept of gene therapy, highlighting its potential to treat genetic disorders by altering genetic makeup. It addresses misconceptions about 'designer babies' and emphasizes the focus on treating diseases. Various types of gene therapy, particularly those involving viral vectors, are discussed. The video delves into fetal gene therapy, its ethical considerations, and recent research, including promising studies on mice. It also touches on the future of gene editing, potential ethical dilemmas, and the importance of guidelines. The video concludes with resources for further learning.

What is a common misconception about gene therapy?

• It can cure all diseases.

• It is a form of traditional medicine.

• It is only used for cosmetic purposes.

• It will be used to create 'designer babies'.

How do scientists use viruses in gene therapy?

• They use viruses to destroy harmful cells.

• They use viruses to deliver genetic instructions.

• They use viruses to create new cells.

• They use viruses to enhance immune response.

What is the primary goal of fetal gene therapy?

• To enhance physical traits.

• To change eye color.

• To prevent diseases before birth.

• To alter intelligence levels.

What criteria did the UK's Gene Therapy Advisory Committee set for fetal treatments?

• They should be available to everyone.

• They should have a clear advantage over other treatments and be for life-threatening diseases.

• They should be used for cosmetic enhancements.

• They should be cheaper than other treatments.

What was a significant finding in the 2018 Nature Medicine study on fetal gene therapy?

• It cured all types of Gaucher's disease.

• It showed potential in treating a disease similar to Gaucher's in mice.

• It was unsuccessful in all animal models.

• It led to severe side effects in mice.

What is a major concern when starting clinical trials for fetal gene therapy?

• The cost of the trials.

• The potential impact on a person's life.

• The availability of resources.

• The speed of the trials.

What was controversial about the Chinese researcher's CRISPR trial?

• It was done in collaboration with multiple countries.

• It was the first of its kind.

• It was highly successful.

• It was done without ethical approval.

What is a potential future concern with gene editing?

• The speed of gene editing.

• The possibility of edited genes being passed to offspring.

• The cost of gene editing.

• The lack of interest in gene editing.

Why are guidelines important in gene therapy research?

• To limit the number of researchers involved.

• To speed up the research process.

• To maintain ethical standards and safety.

• To ensure the research is cost-effective.

What is the current primary intention of fetal gene therapy?

• To increase intelligence.

• To enhance physical appearance.

• To provide necessary treatments.

• To alter personality traits.