Inhibitors of the ATPase p97/VCP: From basic research to clinical applications

S Kilgas, K Ramadan - Cell Chemical Biology, 2023 - cell.com
Protein homeostasis deficiencies underlie various cancers and neurodegenerative
diseases. The ubiquitin-proteasome system (UPS) and autophagy are responsible for most …

[HTML][HTML] Valosin-containing protein (VCP)/p97: a prognostic biomarker and therapeutic target in cancer

S Costantini, F Capone, A Polo, P Bagnara… - International journal of …, 2021 - mdpi.com
Valosin-containing protein (VCP)/p97, a member of the AAA+ ATPase family, is a molecular
chaperone recruited to the endoplasmic reticulum (ER) membrane by binding to membrane …

Therapeutic developments for valosin-containing protein mediated multisystem proteinopathy

V Boock, B Roy, G Pfeffer… - Current Opinion in …, 2023 - journals.lww.com
Being a rare disease, it is challenging to perform large-scale randomized control trials
(RCTs) in VCP-MSP. However, it is important to recognize potential therapeutic targets, and …

[HTML][HTML] Pharmaceutical patent landscaping: A novel approach to understand patents from the drug discovery perspective

Y Gadiya, P Gribbon, M Hofmann-Apitius… - Artificial Intelligence in the …, 2023 - Elsevier
Patents play a crucial role in the drug discovery process by providing legal protection for
discoveries and incentivising investments in research and development. By identifying …

[HTML][HTML] A combination treatment based on drug repurposing demonstrates mutation-agnostic efficacy in pre-clinical retinopathy models

H Leinonen, J Zhang, LM Occelli, U Seemab… - Nature …, 2024 - nature.com
Inherited retinopathies are devastating diseases that in most cases lack treatment options.
Disease-modifying therapies that mitigate pathophysiology regardless of the underlying …

[HTML][HTML] Molecular mechanisms driving and regulating the AAA+ ATPase VCP/p97, an important therapeutic target for treating cancer, neurological and infectious …

S Valimehr, A Sethi, M Shukla, S Bhattacharyya… - Biomolecules, 2023 - mdpi.com
p97/VCP, a highly conserved type II ATPase associated with diverse cellular activities (AAA+
ATPase), is an important therapeutic target in the treatment of neurodegenerative diseases …

[HTML][HTML] VCP/p97 inhibitor CB-5083 modulates muscle pathology in a mouse model of VCP inclusion body myopathy

C Cheng, L Weiss, H Leinonen, A Shmara… - Journal of Translational …, 2022 - Springer
Background Pathogenic gain of function variants in Valosin-containing protein (VCP) cause
a unique disease characterized by inclusion body myopathy with early-onset Paget disease …

[HTML][HTML] Exploring in vivo combinatorial chemo-immunotherapy: Addressing p97 suppression and immune reinvigoration in pancreatic cancer with tumor …

YL Lo, CY Li, TF Chou, CP Yang, LL Wu… - Biomedicine & …, 2024 - Elsevier
Pancreatic ductal adenocarcinoma (PDAC) has an extremely devastating nature with poor
prognosis and increasing incidence, making it a formidable challenge in the global fight …

[HTML][HTML] Retinoid therapy restores eye-specific cortical responses in adult mice with retinal degeneration

CYL Huh, H Leinonen, T Nakayama, JR Tomasello… - Current Biology, 2022 - cell.com
Despite the recent emergence of multiple cellular and molecular strategies to restore vision
in retinal disorders, it remains unclear to what extent central visual circuits can recover when …

[HTML][HTML] An integrated in silico, in vitro and tumor tissues study identified selenoprotein S (SELENOS) and valosin-containing protein (VCP/p97) as novel potential …

S Costantini, A Polo, F Capone, M Accardo, A Sorice… - Cancers, 2022 - mdpi.com
Simple Summary Triple negative breast cancer (TNBC) represents a clinical challenge
because its early relapse, poor overall survival and lack of effective treatments. Altered …