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@ARTICLE{Mileti:298895,
author = {N. Miletić and J. Weckesser and T. Mosler and R. Rathore
and M. E. Hoffmann and P. Gehrtz and S. Schlesiger and I. V.
Hartung and N. Berner$^*$ and S. Wilhelm and J. Müller and
B. Adhikari and V. Němec and S. A. Sivashanmugam and L.
Elson and H. Holzmann and M. P. Schwalm and L. Hoffmann and
K. R. Abdul Azeez and S. Müller and B. Kuster$^*$ and E.
Wolf and I. Đikić and S. Knapp$^*$},
title = {{W}orkflow for {E}3 {L}igase {L}igand {V}alidation for
{PROTAC} {D}evelopment.},
journal = {ACS chemical biology},
volume = {20},
number = {2},
issn = {1554-8929},
address = {Washington, DC},
publisher = {Soc.},
reportid = {DKFZ-2025-00338},
pages = {507-521},
year = {2025},
note = {2025 Feb 21;20(2):507-521},
abstract = {Proteolysis targeting chimeras (PROTACs) have gained
considerable attention as a new modality in drug discovery.
The development of PROTACs has been mainly focused on using
CRBN (Cereblon) and VHL (Von Hippel-Lindau ligase) E3 ligase
ligands. However, the considerable size of the human E3
ligase family, newly developed E3 ligase ligands, and the
favorable druggability of some E3 ligase families hold the
promise that novel degraders with unique pharmacological
properties will be designed in the future using this large
E3 ligase space. Here, we developed a workflow aiming to
improve and streamline the evaluation of E3 ligase ligand
efficiency for PROTAC development and the assessment of the
corresponding 'degradable' target space using broad-spectrum
kinase inhibitors and the well-established VHL ligand VH032
as a validation system. Our study revealed VH032 linker
attachment points that are highly efficient for kinase
degradation as well as some of the pitfalls when using
protein degradation as a readout. For instance, cytotoxicity
was identified as a major mechanism leading to PROTAC- and
VHL-independent kinase degradation. The combination of E3
ligase ligand negative controls, competition by kinase
parent compounds, and neddylation and proteasome inhibitors
was essential to distinguish between VHL-dependent and
-independent kinase degradation events. We share here the
findings and limitations of our study and hope that this
study will provide guidance for future evaluations of new E3
ligase ligand systems for degrader development.},
cin = {FM01 / MU01},
ddc = {540},
cid = {I:(DE-He78)FM01-20160331 / I:(DE-He78)MU01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39932098},
doi = {10.1021/acschembio.4c00812},
url = {https://inrepo02.dkfz.de/record/298895},
}