ProjektTargetVPC – Towards clinical translation of mutation-independent treatment of RD by inhibiting VCP
Grunddaten
Akronym:
TargetVPC
Titel:
Towards clinical translation of mutation-independent treatment of RD by inhibiting VCP
Laufzeit:
01.07.2023 bis 30.06.2025
Abstract / Kurz- beschreibung:
To date, almost all forms of retinal degeneration (RD) are untreatable and lead to visual impairment and even blindness. Based on significant experimental data, we have reason to believe that disturbances in the dynamic regulation of a functional proteome (the proteostasis) and energy deficiency are common features of RD. Given the high energy demands of photoreceptors, chronic disturbances of cellular protein and metabolic homeostasis inevitably lead to dysfunction, energy deficiency and eventually their degeneration. We have identified "valosin-containing protein" (VCP), an enzyme of energy metabolism (ATPase) essential for the very energy-consuming process of degrading misfolded proteins, as an important therapeutic target.
Restoring the balance of proteostasis by inhibiting VCP with three VCP inhibitors improved photoreceptor viability and function in different animal models of RD. We now aim to further develop this preclinical proof of concept to identify and validate a superior VCP-targeting drug for future human clinical use.
Restoring the balance of proteostasis by inhibiting VCP with three VCP inhibitors improved photoreceptor viability and function in different animal models of RD. We now aim to further develop this preclinical proof of concept to identify and validate a superior VCP-targeting drug for future human clinical use.
Beteiligte Mitarbeiter/innen
Leiter/innen
Department für Augenheilkunde
Kliniken und klinische Institute, Medizinische Fakultät
Kliniken und klinische Institute, Medizinische Fakultät
Ansprechpartner/innen
Department für Augenheilkunde
Kliniken und klinische Institute, Medizinische Fakultät
Kliniken und klinische Institute, Medizinische Fakultät
Lokale Einrichtungen
Forschungsinstitut für Augenheilkunde
Department für Augenheilkunde
Kliniken und klinische Institute, Medizinische Fakultät
Kliniken und klinische Institute, Medizinische Fakultät
Geldgeber
Toronto, Kanada