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@ARTICLE{Bochicchio:829374,
author = {Bochicchio, Anna and Jordaan, Sandra and Losasso, Valeria
and Chetty, Shivan and Casasnovas Perera, Rodrigo and
Ippoliti, Emiliano and Barth, Stefan and Carloni, Paolo},
title = {{D}esigning the {S}niper: {I}mproving {T}argeted {H}uman
{C}ytolytic {F}usion {P}roteins for {A}nti-{C}ancer
{T}herapy via {M}olecular {S}imulation},
journal = {Biomedicines},
volume = {5},
number = {1},
issn = {2227-9059},
address = {Basel},
publisher = {MDPI},
reportid = {FZJ-2017-03087},
pages = {9},
year = {2017},
abstract = {Targeted human cytolytic fusion proteins (hCFPs) are
humanized immunotoxins for selective treatment of different
diseases including cancer. They are composed of a ligand
specifically binding to target cells genetically linked to a
human apoptosis-inducing enzyme. hCFPs target cancer cells
via an antibody or derivative (scFv) specifically binding to
e.g., tumor associated antigens (TAAs). After
internalization and translocation of the enzyme from
endocytosed endosomes, the human enzymes introduced into the
cytosol are efficiently inducing apoptosis. Under in vivo
conditions such enzymes are subject to tight regulation by
native inhibitors in order to prevent inappropriate
induction of cell death in healthy cells. Tumor cells are
known to upregulate these inhibitors as a survival mechanism
resulting in escape of malignant cells from elimination by
immune effector cells. Cytosolic inhibitors of Granzyme B
and Angiogenin (Serpin P9 and RNH1, respectively), reduce
the efficacy of hCFPs with these enzymes as effector
domains, requiring detrimentally high doses in order to
saturate inhibitor binding and rescue cytolytic activity.
Variants of Granzyme B and Angiogenin might feature reduced
affinity for their respective inhibitors, while retaining or
even enhancing their catalytic activity. A powerful tool to
design hCFPs mutants with improved potency is given by in
silico methods. These include molecular dynamics (MD)
simulations and enhanced sampling methods (ESM). MD and ESM
allow predicting the enzyme-protein inhibitor binding
stability and the associated conformational changes,
provided that structural information is available. Such
“high-resolution” detailed description enables the
elucidation of interaction domains and the identification of
sites where particular point mutations may modify those
interactions. This review discusses recent advances in the
use of MD and ESM for hCFP development from the viewpoints
of scientists involved in both fields.},
cin = {IAS-5 / INM-9},
ddc = {610},
cid = {I:(DE-Juel1)IAS-5-20120330 / I:(DE-Juel1)INM-9-20140121},
pnm = {899 - ohne Topic (POF3-899)},
pid = {G:(DE-HGF)POF3-899},
typ = {PUB:(DE-HGF)16},
UT = {WOS:000398714600009},
pubmed = {pmid:28536352},
doi = {10.3390/biomedicines5010009},
url = {https://juser.fz-juelich.de/record/829374},
}