Do discussions about gene therapy leave you scratching your head? Well look no further- the following video will guide you through how genes work- as blueprints that determine everything about the body, including how well you clot – and why gene therapy can be used to create new therapies for hemophilia.
‘Jean’ explains how gene therapy works to overcome the mutations in genes, such as the genes that produce clotting factor. Scientists use vectors- viruses that have been emptied of the part that makes people sick- to introduce functional genes into the body. And once these functional genes are reproduced, they start to make effective clotting proteins in cells. Curious to know more? Click here to watch the full NHF video “What is Gene Therapy?”