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HMS Professor Announces Promising Gene Therapy Results

For example, one viral technique involves introducing a changed gene that can infect all cells in the body, not just targeted organs.

In men, this kind of virus could reach the testis and lead to "germ-line transmission," an effect which the National Institute of Health has deemed negative, said White.

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White said Roth's findings are important in both the fields of hemophilia and gene therapy research.

He said after the therapy some of the patients in the study had higher levels of the needed Factor VIII, which is necessary for blood clotting, than before receiving the gene therapy.

"His study did achieve Factor VIII to levels of 2 to 4 percent in some patients," White said. "These people progressed from severe hemophiliacs to moderately severe hemophiliacs."

Currently, hemophiliacs can use bio-engineered Factor VIII to treat themselves, but at very high cost, White said.

The other traditional method for treatment involves blood products containing concentrates of plasma proteins. But this treatment has also carried viruses such as HIV and hepatitis.

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