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Chapter: Biotechnology Applying the Genetic Revolution: Gene Therapy

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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.

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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