Gene Therapy Flashcards
How many chromosomes do humans have?
22 + xy or xx
What is germ-line gene therapy?
What is somatic gene therapy?
What is exvivo gene therapy/
What is stable gene trasnfer?
Is obtained when the therapeutic gene integrates into host cell chromosomal DNA
When episomal vectors are used for gene transmittance what is required?
Original of replication
What is ADA?
Adenosine deaminase deficiency is where individuals are severely immunodeficienttherefore require bone marrow transplant or administration of the ADA protein
How often do individauls require ADA protein?
1 to 2 month intervals
What is familial hypercholesterolemia?
The most commonly employed approaches for cancer gene therapy influcde?
What is the bystander effect?
Cerepro what is it?
brain tumor treatment
What is the MDR-1 gnee/
What is luxturna?
Treatment for inherited genetic mutation that is associated with retinal dystrophy (1 time treatment), Progressive loss of peripheral and central vision
What gene does luxturna target?
RPE65 gene (Eye Target)
What is lymfactin?
Gene transfer vector carrying the human VEGF-C gene. Where it promotes the body to produce VEGFC to fix damage to area
What are the limitations of gene therapy?
Short lives
Immune repsonse
Viral Vectors
Multigene disorders
May induce tumors
What are the deliveries of non-viral tene transfer
Injection of naked DNA
particle bombardment
Entrapping DNA into liposomes
What viruses can be used for gene transfer?
What are the ideal vector for gene transfer?
What is Lyfengia?
Cell based gene therapy that uses a lentiviral vector
WHat is Vyjuvek?
Used to treat EB (Butterfly skin)
