Gene Replacement Therapies

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Welcome to Gene Replacement Therapies.  Gene Replacement Therapies are the medical insertion, alteration, or removal of genes within an individual’s cells and biological tissues to treat disease. It is a technique for correcting defective genes that are responsible for disease development. The most common form of gene therapy involves the insertion of functional genes into an unspecified genomic location in order to replace a mutated gene, but other forms involve directly correcting the mutation or modifying normal gene that enables a viral infection. Although the technology is still in its infancy, it has been used with some success.

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Gregg Semenza of Johns Hopkins Medicine on Science

Gregg Semenza, MD, Ph.D., of the Johns Hopkins McKusick-Nathans Institute of Genetic Medicine on the excitement and unpredictability of being a scientist.