Translational perspectives in HTRA1-associated disorders: biomarkers, gene therapy, and future directions

High-temperature requirement A serine protease 1 (HTRA1) is an enzyme important for vascular and tissue homeostasis, with mutations linked to cerebral small vessel disease and systemic conditions like age-related macular degeneration and osteoarthritis. These mutations can lead to microvascular damage and symptoms such as strokes, cognitive decline, and alopecia. Diagnosis involves neuroimaging, biomarkers, and genetic testing. Emerging treatments, including gene therapy and small-molecule modulators, show promise for addressing these disorders. The review highlights the importance of understanding molecular mechanisms and developing precision medicine strategies for HTRA1-associated conditions.