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

Research output: Contribution to journalArticle

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 languageEnglish (US)
Pages (from-to)622-634.e6
JournalCell Chemical Biology
Volume27
Issue number5
DOIs
StatePublished - May 21 2020

Keywords

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

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Medicine
  • Molecular Biology
  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry

Fingerprint Dive into the research topics of 'Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones'. Together they form a unique fingerprint.

  • Cite this