Drug intelligence / Profile preview

Granulin

Development stage
Preclinical
Modality
Recombinant Proteins and Enzymes, Peptides
Administration
Intrathalamic, Intra-cisterna Magna, Intravenous
01

Overview

Granulin, also known as progranulin (PGRN), is a multifunctional protein involved in various biological processes including lysosomal function, neuroinflammation, and neuronal survival. It is the precursor to several granulin motifs. Mutations in the GRN gene, which encodes progranulin, are a significant cause of hereditary Frontotemporal Dementia (FTD) due to reduced progranulin levels. Therapeutically, granulin or its derived peptides are being investigated for their potential to restore progranulin levels in FTD or for applications like wound healing. Conversely, elevated progranulin levels are associated with increased malignancy in tumors and are being studied as a biomarker for conditions such as prediabetes and diabetes.

Other names
ProgranulinPGRNGRN
02

Targets

HEXA (Hexosaminidase subunit alpha)CTSD (Cathepsin D)GBA1 (Glucosylceramidase)

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