Gene‐targeted therapies: Overview and implications

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.

Note from the editors

“…(Yu et al, 2023), provides an overview of the current landscape of gene‐targeted therapies and their potential, presenting a rationale for the use of such therapies in a more systematic manner, while recognizing some of the serious ethical, financial, and infrastructure considerations of widespread adoption of such therapies. Gene‐targeted therapies: Overview and implications (Brooks, Urv, & Parisi, 2023) is a primer for readers that provides a general overview of the three main categories of gene‐targeted therapies, including gene therapy, gene editing, and oligonucleotide therapies.…”