David R. Liu: Can we cure genetic diseases by rewriting DNA?

The video discusses the significance of DNA and the impact of point mutations on genetic diseases. It introduces CRISPR technology and the development of base editing, a method to correct point mutations without disrupting the genome. The video explains the mechanism of base editors, their advancements, and potential applications in treating genetic diseases and improving agriculture. It highlights the challenges and future prospects of base editing, emphasizing the need for ethical considerations.

What is the most common type of change in DNA that can lead to genetic diseases?

• Insertion of new DNA sequences

• Duplication of genes

• Deletion of DNA segments

• Point mutations

Which genetic disease is caused by a single point mutation in the hemoglobin gene?

• Tay-Sachs disease

• Muscular dystrophy

• Sickle cell anemia

• Progeria

What is the primary function of CRISPR in bacteria?

• To replicate DNA

• To fight viral infections

• To produce proteins

• To repair damaged cells

How do base editors differ from CRISPR scissors?

• Base editors are naturally occurring, unlike CRISPR scissors

• Base editors convert one base to another without cutting DNA

• Base editors cut DNA, while CRISPR scissors do not

• Base editors disrupt genes, while CRISPR scissors repair them

What challenge did researchers face when developing a base editor to convert A to G?

• Difficulty in disabling CRISPR scissors

• Inability to target specific DNA sequences

• Incompatibility with human cells

• Lack of a naturally occurring protein to perform the conversion

Which animal was used to demonstrate the correction of progeria using base editing?

• Cat

• Dog

• Mouse

• Rabbit

What is one potential application of base editing in agriculture?

• Increasing crop yield through genetic modification

• Developing pest-resistant plants

• Introducing single-DNA letter changes for better crops

• Enhancing photosynthesis efficiency

What is a major challenge in delivering base editors to human cells?

• Ensuring they do not cause immune reactions

• Finding suitable viral vectors for delivery

• Achieving high precision in editing

• Minimizing off-target effects

Why is collaboration with ethicists important in the development of base editing?

• To apply the technology safely and ethically

• To maximize the speed of research

• To increase public awareness

• To ensure the technology is cost-effective

What is the ultimate goal of base editing in the context of genetic diseases?

• To enhance human genetic traits

• To replace traditional gene therapy

• To provide a means to protect and repair DNA

• To completely eliminate all genetic diseases