Gene Therapy

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