Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones

Prerana Ramadurgum; Da Nae R. Woodard; Steffi Daniel; Hui Peng; Prema L. Mallipeddi; Hanspeter Niederstrasser; Melina Mihelakis; Viet Q. Chau; Peter M. Douglas; Bruce A. Posner; John D. Hulleman

doi:10.1016/j.chembiol.2020.03.006

Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones

Prerana Ramadurgum, Da Nae R. Woodard, Steffi Daniel, Hui Peng, Prema L. Mallipeddi, Hanspeter Niederstrasser, Melina Mihelakis, Viet Q. Chau, Peter M. Douglas, Bruce A. Posner, John D. Hulleman

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8 Scopus citations

Abstract

Ramadurgum et al. identify 17 new stabilizers of the E. coli dihydrofolate reductase (ecDHFR) destabilizing domain (DD) and validate them both in vitro and in vivo (mouse ocular and hepatic tissue). They also demonstrate the ability of these compounds to simultaneously control endogenous (stabilizer-defined) and user-defined (ecDHFR-DD-based) pathways.

Original language	English (US)
Pages (from-to)	622-634.e6
Journal	Cell Chemical Biology
Volume	27
Issue number	5
DOIs	https://doi.org/10.1016/j.chembiol.2020.03.006
State	Published - May 21 2020
Externally published	Yes

Bibliographical note

Publisher Copyright:
© 2020 Elsevier Ltd

Keywords

E. coli dihydrofolate reductase
chemical biology
conditional regulation
destabilizing domain
dual use
gene therapy
high-throughput screening
ocular
pharmacological chaperone

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1016/j.chembiol.2020.03.006

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Ramadurgum, P., Woodard, D. N. R., Daniel, S., Peng, H., Mallipeddi, P. L., Niederstrasser, H., Mihelakis, M., Chau, V. Q., Douglas, P. M., Posner, B. A., & Hulleman, J. D. (2020). Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones. Cell Chemical Biology, 27(5), 622-634.e6. https://doi.org/10.1016/j.chembiol.2020.03.006

Ramadurgum, P, Woodard, DNR, Daniel, S, Peng, H, Mallipeddi, PL, Niederstrasser, H, Mihelakis, M, Chau, VQ, Douglas, PM, Posner, BA & Hulleman, JD 2020, 'Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones', Cell Chemical Biology, vol. 27, no. 5, pp. 622-634.e6. https://doi.org/10.1016/j.chembiol.2020.03.006

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abstract = "Ramadurgum et al. identify 17 new stabilizers of the E. coli dihydrofolate reductase (ecDHFR) destabilizing domain (DD) and validate them both in vitro and in vivo (mouse ocular and hepatic tissue). They also demonstrate the ability of these compounds to simultaneously control endogenous (stabilizer-defined) and user-defined (ecDHFR-DD-based) pathways.",

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doi = "10.1016/j.chembiol.2020.03.006",

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AU - Peng, Hui

AU - Mallipeddi, Prema L.

AU - Niederstrasser, Hanspeter

AU - Mihelakis, Melina

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AU - Posner, Bruce A.

AU - Hulleman, John D.

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Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones

Abstract

Bibliographical note

Keywords

UN SDGs

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