AM L5.2 Gene therapy Flashcards
Two gene therapy options
Knock down
or
knock in
(i.e. gain of function or silencing)
Major obstacle in gene therapy
Delivery of the gene into the cell
Two delivery options
Liposomes
Viral vectors
Liposomes or viral vectors are long term
Viral vectors
Liposomes or viral vectors are potentially cytotoxic?
Both
Liposomes or viral vectors have high level of gene expression?
Viral vectors
Liposomes or viral vectors are taken up by endocytosis?
Liposomes
Liposomes or viral vectors have a range of expression efficiencies?
Liposomes
Liposomes or viral vectors are more likely to trigger immune response?
Viral vectors
Viral vectors have been used to increase HDL in mice by…
Expression of human apoA1
Familial hyperglycemia has initially been treated with gene therapy but…
response is not sustained - retroviral silencing
Lipoprotein lipase deficiency symptoms
abdominal pain, fatty deposits in the skin and retina
Fatal pancreatitis, diabetes, CVD
First treatment for Lipoprotein lipase deficiency
Glybera
Autosomal-recessive disease that has been treated with gene therapy?
Lipoprotein lipase deficiency
