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Gene Therapy
The vast majority of rare genetic disorders are currently without any cure, causing misery to an estimated 70 million individuals in our country. Gene therapy offers a potential platform technology to find cures for most genetic disorders, particularly those due to defects in a single gene. Moreover, gene therapy holds treatment potential in many cancers and other disorders, such as peripheral artery diseases and arthritis. In many diseases, it offers the only hope towards a cure.
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