The Second Coming of Gene Therapy

The Second Coming of Gene Therapy.

1)  With what disease was Katlyn diagnosed?

2)  What specifically, caused Katlyn’s disease?

3)  What were two treatment options for Katlyn?

4)  What is a retrovirus?

5)  What did the Gene Therapy do for Katlyn (specifically)

Dark Days

1)  What problem did James Wilson try to solve with gene therapy?

2)  What are the problems with using a virus as a vector for gene therapy?

3)  Wilsons first attempt to solve the problem he engineered a retrovirus called___.

Lighting the way

1)  Which gene mutation can cause Leber congenital amaurosis?

2)  Which virus was used to inject the healthy gene?

3)  Where was the virus injected.

4)  What is hemophilia?

5)  What defected genes cause hemophilia?

6)  What happened when human test subjects were injected with the AAV virus to treat hemophilia?

7)  What made the eye a good organ to accept the AAV virus for gene therapy?

The cancer connection

1)  Which retrovirus may be use for cancer treatments?

2)  A rare form of blood cancer is caused when B cells become infected with ______.

3)  How does AdV help in treating cancer?