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@PHDTHESIS{Knoll:278045,
author = {Knoll, Rainer},
title = {{O}mics-driven {D}rug {R}epurposing and {T}reatment
{A}ssessment in {H}uman {V}iral {D}iseases},
school = {Rheinische Friedrich-Wilhelms-Universität Bonn},
type = {Dissertation},
reportid = {DZNE-2025-00551},
pages = {57 p.},
year = {2025},
note = {Dissertation, Rheinische Friedrich-Wilhelms-Universität
Bonn, 2025},
abstract = {In this cumulative thesis, I present my research on
transcriptomic-based drug repurposing in human viral
infections describing an optimized workflow for drug
prediction, in vitro validation and in vivo studies in
clinical cohorts based on three publications.In the first
publication, I took lead in a larger team effort to
introduce a newly designed drug repurposing approach based
on whole blood transcriptomics data and drug signatures
databases, which was applied to identify potential drug
candidates for treatment of patients across COVID-19
severity groups stratified based on clinical parameters and
transcriptomic phenotypes (Aschenbrenner et al. 2021). One
of the drug candidates identified using this approach was
dexamethasone, which was predicted to be effective in the
most severe group of COVID-19 patients.In the second
publication, I present my findings on transcriptomic
alterations in the monocyte compartment in chronically
infected HIV patients using multi-omics technologies,
demonstrate that these alterations originate from a certain
disease state and identify potential drug candidates for the
reversal of the disease signatures in monocytes (Knoll et
al. 2023). In this study, I further extend the
transcriptomics drug repurposing approach by refining the
underlying disease signatures using single-cell omics for
drug prediction and I validate promising drug candidates
using in vitro stimulation experiments. Reading out direct
drug-induced transcriptional alterations from these in vitro
studies substantially strengthened the results from the drug
repurposing approach.In the third publication, I describe
our framework on how to investigate repurposed drugs in
clinical cohorts in vivo using single-cell transcriptomics
towards precision medicine, exemplified with dexamethasone
treatment in COVID-19 (Knoll et al. 2024). Dexamethasone
caused strong transcriptional and immunomodulatory changes
with a reversal of dysregulation in severe COVID-19
monocytes compared to treatment-naïve patients. Moreover, a
treatment-specific monocyte response state was identified
which stratified outcome and enabled prediction of treatment
responses, stressing the potential of single-cell
transcriptomics for companion diagnostics and mechanistic
studies of repurposed drugs.In conclusion, the research
presented in this thesis describes the design and
application of a transcriptomics-based drug repurposing
pipeline for human viral infections. It highlights the
significant potential of drug repurposing in context of
data-driven disease severity stratification using optimized
cell-state specific disease signatures. Moreover, it
underscores the importance of in vitro validation for
promising drug candidates to reverse disease signatures.
This paves the way for a standardized analytical approach to
evaluate drug indications in clinical cohorts in vivo,
utilizing single-cell transcriptomics for treatment response
stratification, ultimately enabling precision medicine.},
cin = {AG Aschenbrenner / AG Schultze},
cid = {I:(DE-2719)5000082 / I:(DE-2719)1013038},
pnm = {354 - Disease Prevention and Healthy Aging (POF4-354)},
pid = {G:(DE-HGF)POF4-354},
typ = {PUB:(DE-HGF)11},
urn = {urn:nbn:de:hbz:5-82529},
url = {https://pub.dzne.de/record/278045},
}