Specific genetic modifications in the CNS - Cell specific expression and gene specific regulation
Popular Abstract in Swedish Vid genterapi för man in nya gener i en individs celler. Detta kan göras med hjälp av modifierade virus. Dessa virus kan föra in den nya genen, den såkallade transgenen, in i värdcellen men inte bilda nya viruspartiklar. I många fall är det viktigt att begränsa i vilka celler som transgenen uttrycks, då det producerade proteinet kan ge bieffekter i andra celltyper. Det Gene therapy is a promising therapeutic tool for many diseases of the CNS. Lentiviral vectors are particularly attractive since this vector type can transduce both dividing and non-dividing cells, have a relatively large cloning capacity, can sustain long-term transgene expression and have low immunogenicity. The specificity of the vector is however important to avoid potential off-target effects