GM2 Gangliosidosis and Gene Therapy Quiz

What is the primary cause of Tay-Sachs disease?

• A mutation in the HexA gene

• A mutation in the HexB gene

• A mutation in the GM2 gene

• A mutation in the HexC gene

Which of the following is NOT a symptom of GM2 gangliosidosis?

• Seizures

• Difficulty breathing

• Improved brain function

• Loss of developmental skills

What is the main goal of gene therapy for GM2 gangliosidosis?

• To remove the HexA and HexB genes

• To eliminate all viral vectors

• To deliver working HexA and HexB genes

• To increase the production of GM2 gangliosides

How are therapeutic genes delivered to the brain or spinal cord in gene therapy?

• Through dietary supplements

• By surgical implantation

• Via a single injection using viral vectors

• Through oral medication

What is a necessary step in the research process for gene therapies?

• Direct marketing to patients

• Approval by a single scientist

• Clinical trials to test safety and efficacy

• Immediate public distribution