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Gene Therapies

Gene therapy studies underway for treatment of inherited hematological disorders

Published in Gene Therapy Weekly, July 20th, 2006

Gene therapy studies are underway for the treatment of inherited hematological disorders.

According to a recently published report from the United States, "Gene therapy, a molecular medicine based on vector-mediated transfer of therapeutic genes, holds promise for a cure of monogenetic inherited diseases.

"In recent years, tremendous progress has been reported in the treatment of hematological disorders: clinical trials in severe combined immune deficiencies have been successful by using retroviral vectors to express target genes in hematopoietic stem cells, which after transplantation efficiently reconstituted the immune system concomitant with...

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