GEN #19 Gene Therapy II

a _______ mutation is the easiest to treat

The ______ the size of DNA to be introduced the more difficult in general.

________ levels vary in response to metabolic or physiological mediators

target tissues that are the most difficult to target are _______

ADA is an enzyme in the

ADA stands for

________ are particularly sensitive to decreased ADA activity because they rely heavily on the ________________.

ADA is characterized by a __________ immunodeficiency syndrome

CFTR stand for

The most common CF defects is ______

The CFTR gene os located on the

AAV stands for

The first gene therapy trial used an adenoviral vector in which ________ and _______ had been deleted.

The three non-viral vector systems are

Wiskott-Aldrich Syndrome is an

WAS is characterized by

WAS gene codes for protein that regulates _________ in immune cells.

WAS stands for

Ex vivo approach to gene therapy for WAS using a retroviral
vector obtained ___________ by ___________

WAS-expressing retroviral vector composed of the ________ backbone virus pseudotyped with the gibbon ape ________ virus envelop protein.

In the PD clinical trial the AAV vector contained the gene for ___________ which is required for __________ biosynthesis

The vector for PD patients was injected into the ________ region of the brain