Gene Therapy

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Gene Therapy

Normal alleles of gene inserted into target cells using Genetically modified Virus or liposomes (spherical phospholipid bilayers) 

Normal form of gene is transcribed and translated

a functioning protein is produced in target cells. (in CF gene CFTR protein is produced and incorparated into cell membrane, restoring ion channel and avoiding symptoms of CF)

Genetically Modified virus

- DNA that allows it to replicate is removed - replaced with normal allele of desired gene along with promotor sequence that initicates transcription/translation of gene. 

in CF viral DNA remains independent within nucleus of our cells

X causes inflamatory response with symptoms such as headache fever and raised heart rate. 


- Copy of normal allele inserted into loop of DNA - called a plasmid

- positively charged head groups of phospholipids combine with DNA to forn liposome-DNA complex

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