Module 12 Flashcards
enzyme replacement therapy
recombinant enzyme infusion
substrate reduction therapy
oral drug reduces substrate so enzyme can function more effectively
pharmacological chaperone therapy
oral drug binds to misfolded protein, restoring function
gene therapy
therapy uses genes directly or targets genes
direct delivery
therapeutic gene packaged into delivery vehicle and injected
cell-based delivery
therapeutic gene packaged into a vehicle, introduced to in vitro stem cell, reintroduced to patient
germline gene therapy
alters DNA of fertilized egg for all cells to be affected. not done in humans
somatic gene therapy
only somatic cells, not passed down to generations
transgenic organisms
combination of DNA from different organisms
in vivo
delivered directly to patient
ex vivo
delivered to cell then to patient
gene therapy vectors
retrovirus, adenovirus, naked DNA, liposome plasmid vector
tropism
affinity for cell or tissue type
severe combined immune deficiency
deficiency of enzyme ADA leading to toxic buildup of deoxy ATP destroying T/B cells
notorious setbacks
19yo with enzyme deficiency toxic buildup of ammonia died after gene therapy; 4 French kids developed leukemia
clinicaltrials.gov
list of all gene therapy trials ongoing
gene replacement/augmentation therapy
adds functional copy of a gene
end-run therapy
target is downstream of a gene that supplies a needed function
suppression therapy
targets GOF mutations to stop a bad gene. gene silencing RNAi
gene supplementation therapy
increases gene dosage
cancer therapy
targets tumor cells while leaving healthy cells
CAR T cell therapy
engineered t/b-cell combination, super t-cell
gene-based therapy
using genes to produce therapeutic agent
ZFN
zinc finger nuclease cuts one gene at a time
