CYSTIC
FIBROSIS GENE THERAPY BY ADENOVIRUS
Because the lungs are relatively
accessible to viral infection, cystic fibrosis
has been a prime candidate for gene therapy. The healthy version of the
cystic fibrosis gene has been cloned and inserted into a crippled adenovirus.
Aerosols containing the engineered adenovirus with the cystic fibrosis gene
have been sprayed into the noses and lungs, first of rats, and then of humans.
In some instances the healthy cystic fibrosis gene was expressed and normal
chloride ion movements were also restored. Unfortunately, expression falls off
over a 30-day period and repeated doses of virus have little effect, largely
because of recognition and destruction of the virus by the immune system.
It is hoped that in the near
future, improved vectors will allow cystic fibrosis to be cured by nasal sprays
containing genetically engineered viruses. Note, however, that this sort of
gene therapy cures only the symptoms in the lungs; it does not correct the
genetic defect in the germline cells. The defect will still be passed on to the
next generation.
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