Gene‐targeted therapies: Overview and implications

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 p...

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Bibliographic Details
Published in:American journal of medical genetics. Part C, Seminars in medical genetics Seminars in medical genetics, 2023-03, Vol.193 (1), p.13-18
Main Authors: Brooks, P J, Urv, Tiina K., Parisi, Melissa A.
Format: Article
Language:English
Subjects:
Genetic Therapy
Genetics
Humans
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Summary: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.
ISSN:1552-4868
1552-4876
1552-4876
DOI:10.1002/ajmg.c.32033