Chapter 11, Problem 3TQ

Summary Introduction

To review:

The advantages and disadvantages of usage of viral vectors for the treatment of haemophilia.

Introduction:

Hemophilia is a genetic disorder characterized by the absence of clotting factors like factor VIII and IX in the blood. They are inherited in an autosomal X-linked pattern. The symptoms of this disorder include profuse bleeding upon minor injury, easy bleeding, and risk of internal bleeding in joints and the brain.