Gene Therapy

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deoxyadenosine (a waste product) into inosine. This leads to the rapid build-up of deoxyadenosine in the system, which becomes phosphorlyzed into a toxic triphosphate, which kills T-cells. The result is an almost complete failure of the immune system and early death.
Previous treatment options included bone marrow transplants, which worked well with matched donors. A major breakthrough occurred with the development of polyethylene glycol coated ADA (PEG-ADA). This treatment introduces coated ADA into the blood stream, although not into the cells. It requires expensive, painful shots on a weekly basis, but it succeeded in giving children with ADA deficiency a new lease on life. While their immune systems were far from normal, PEG-ADA allows some semblance of a normal life and a much-increased life span.
The first patient to undergo federally approved was a young girl named Ashanti DeSilva, in 1990. Ashanti's success immediately sparked a torrent of investment and research. ...

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have predicted that the 21st century will be the "biology century." The analytical power arising from the reference DNA sequences of several entire genomes and other genomic resources is anticipated to help jump-start the new millennium.
The HGP's continued emphasis is on obtaining a complete and highly accurate reference sequence (1 error in 10,000 bases) that is largely continuous across each human chromosome. Scientists believe that knowing this sequence is critically important for understanding human biology and for applications to other fields.
The next step to is to find a vehicle to transport the new genes. This is done through the use of vectors. A vector is a DNA molecule ...

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