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Gene-targeted therapies: Overview and implications |
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| Authors: | P J Brooks Tiina K Urv Melissa A Parisi |
| Institution: | 1. National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland, USA;2. Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA |
| Abstract: | Gene-targeted therapies (GTTs) are therapeutic platforms that are in principle applicable to large numbers of monogenic diseases. The rapid development and implementation of GTTs have profound implications for rare monogenic disease therapy development. This article provides a brief summary of the primary types of GTTs and a brief overview of the current state of the science. It also serves as a primer for the articles in this special issue. |
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