1.11.1 Shaping the framework for development of rare disease therapeutics

Question 1

orphan diseases.

Answer 1

Diseases with poor financial incentives for the private sector to invest in research and development of potential treatment.

Question 2

neglected diseases

Answer 2

Diseases that often affect the world’s poorest people, inflicting a severe health burden.

Question 3

green fluorescent protein (GFP)

Answer 3

Protein isolated from the jellyfish Aequorea victoria that emits green fluorescence and is commonly used as a fluorescent reporter gene in expression constructs.

Question 4

cell membrane

Answer 4

called the plasma membrane in animals.

Question 5

Mitochondria

Answer 5

are the powerhouses of the cell, tiny engines found in most eukaryotic cells convert food and oxygen into the fuel (ATP) that powers all life.

Question 6

membrane potential

Answer 6

like a tiny battery, has a negative voltage inside compared to outside, providing its own electrical energy.

Question 7

heterologous expression

Answer 7

Terms referring to the expression of a gene/protein in an organism or cell that doesn’t naturally contain the gene or its product.