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Updated: Aug 8, 2025

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Gene-targeted therapies: Overview and implications
P J Brooks1, Tiina K Urv1, Melissa A Parisi2
1National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland, USA.
None:
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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