Gene-targeted therapies: Overview and implications.
| Autor: | Brooks PJ; National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland, USA., Urv TK; National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, Maryland, USA., Parisi MA; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA. |
|---|---|
| Jazyk: | angličtina |
| Zdroj: | American journal of medical genetics. Part C, Seminars in medical genetics [Am J Med Genet C Semin Med Genet] 2023 Mar; Vol. 193 (1), pp. 13-18. Date of Electronic Publication: 2023 Mar 02. |
| DOI: | 10.1002/ajmg.c.32033 |
| Abstrakt: | 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. (Published 2023. This article is a U.S. Government work and is in the public domain in the USA. American Journal of Medical Genetics Part C: Seminars in Medical Genetics published by Wiley Periodicals LLC.) |
| Databáze: | MEDLINE |
| Externí odkaz: |
|