Gene Therapy
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8 Terms
what is gene therapy
insertion of a functional DNA sequence to counteract a non-functional gne by producing a functional protein
what is germ line therapy
replacing genes in gametes
is inheritable
somatic cell therapy
targets cells in affected tissues and only affects individual concerned
problems with gene therapy
may introduce other genes
may activate oncogenes which can cause cancer
not permanent
difficult to get new genes into genome of cells
benefit of gene therapy
alleviation of genetic disease symptoms
what happens in Duchenne Muscular Dystrophy
exon/s are deleted in dystrophin gene
interferes with rest of gene assmebly
what drug treats DMD
drisapersen
how does drisapersen treat DMD
means that dystrophin gap is patched up so rest of protein can still be made
known as exon skipping
drisapersen introduces a ‘molecular patch’ over the mutated gene so it can be readable again
Note 
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