Gene‐targeted therapies: Overview and implications
Philip J. Brooks, Tiina K. Urv, Melissa A. Parisi
2023American Journal of Medical Genetics Part C Seminars in Medical Genetics10 citationsDOIOpen Access PDF
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.
Topics & Concepts
DiseaseMedicineComputational biologyEngineering ethicsBiologyEngineeringPathologyCRISPR and Genetic EngineeringVirus-based gene therapy researchPluripotent Stem Cells Research