Network of gene therapy hubs to be set up across UK

The video discusses the translation of lab discoveries into clinical benefits, focusing on the role of the Sheffield hub in manufacturing and delivering treatments for rare genetic diseases. It highlights the potential for gene therapy, the importance of innovation, and the economic benefits of retaining intellectual property in the UK. The video also emphasizes the need for skilled individuals and the focus on rare diseases across various disciplines.

What is one of the main challenges in translating lab discoveries into clinical trials?

• Insufficient funding

• Lack of scientific knowledge

• Limited patient interest

• Absence of manufacturing facilities

Why are genetic rod diseases particularly attractive for gene therapy?

• They are inherited and thus suitable for genetic intervention

• They are easy to diagnose

• They have existing treatments

• They have a high prevalence

How many rare diseases are mentioned as having no effective treatments?

• Over 7000

• Over 9000

• Over 5000

• Over 1000

What is one potential benefit of generating more intellectual property in the UK?

• Decreased research costs

• Increased international collaboration

• Attraction of inward investment and startup creation

• Reduction in disease prevalence

Which of the following is NOT mentioned as a focus area for the center?

• Parkinson's disease

• Diabetes

• Spastic paraplegia

• Motor neuron disease