+ Gene Therapy on the Move Flashcards by A W | Brainscape

Q

What two aims does gene therapy fill (approaches):

A

Delivering functional, therapeutic gene as a substitute for defective / missing one
Reducing levels of harmful defec6tive gene product

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Q

What is Glybera? What does it treat?

A

First European gene therapy licensed
Treats familial lipoprotein lipase deficiency
Recombinant AAV for direct intramuscular injection

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