The disease Leber congenital amaurosis (LCA) caused byloss-of-function mutation of a gene called RPE65, has been treatedwith gene therapy by injecting recombinant AAV vectors containing anormal copy of the RPE65 gene into their retinal epithelial cells.Patients receiving this therapy:
May need to repeat the treatment as the viral DNA isdegraded
May acquire detrimental mutations due to integration of thevirus
Will pass the mutation to their offspring
Now have the normal RPE65 gene
Pharming refers to the use of transgenic animals andplants to produce a protein drug, such as…
GM plants that have enhanced nutritional value
The production of the blood factor antithrombin III in goatmilk
The production of factor VIII protein, the blood-clottingfactor, by bacteria.
A transgenic monkey model for Huntingtondisease
