GEN #19 Gene Therapy II
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a _______ mutation is the easiest to treat
loss of function
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
purine salvage pathway
ADA stands for
________ are particularly sensitive to decreased ADA activity because they rely heavily on the ________________.
- salvage pathway forpurines
ADA is characterized by a __________ immunodeficiency syndrome
combined B & T-cell
CFTR stand for
cystic fibrosis transmembrane conductance regulator
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.
E1 & E3
The three non-viral vector systems are
- Cationic liposomes
- DNA-polymer conjugates
- Naked DNA
Wiskott-Aldrich Syndrome is an
X-linked recessive disease
WAS is characterized by
- Immune deficiency & development of autoimmune diseases
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 ___________
- CD34+ [hematopoietic stem cells]
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
- glutamic acid decarboxylase (GAD)
The vector for PD patients was injected into the ________ region of the brain
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