Understanding Huntington's Disease and Gene Therapy

What is the primary cause of Huntington's disease?

• Mutations in the HTT gene

• Nutritional deficiencies

• Environmental factors

• Viral infections

How does gene therapy aim to help those with Huntington's disease?

• By increasing the production of healthy proteins

• By replacing the entire HTT gene

• By reducing the production of toxic HTT protein

• By removing neurons from the brain

What is a key difference between micro RNAs and antisense oligonucleotides in treating Huntington's disease?

• Antisense oligonucleotides increase toxic protein levels

• Micro RNAs are delivered via vectors

• Antisense oligonucleotides are administered only once

• Micro RNAs require multiple doses

What role do micro RNAs play in gene therapy for Huntington's disease?

• They replace faulty genes

• They bind to and destroy messenger RNA

• They stimulate neuron growth

• They increase the production of toxic proteins

Why might antisense oligonucleotides require multiple doses?

• They are not delivered via vectors

• They need to continuously block the faulty gene

• They are quickly degraded in the body

• They are less effective than micro RNAs