Harry Wilkinson
Designed and tested novel plasmids for correcting COL4A1 mutations using CRISPR editing.
COL4A1-related disease
Understanding and correcting vascular basement membrane disease.
COL4A1-related disorders can affect small blood vessels in the brain and other organs. The project is led by Dr Klaudia Kocsy and focuses on disease mechanisms, experimental models, and therapeutic strategies for COL4A1-related cerebral small vessel disease.
Research focus
From vascular biology to mutation-focused correction strategies.
COL4A1 encodes a collagen IV component of vascular basement membranes. Pathogenic variants can compromise vessel integrity and are associated with a spectrum of cerebrovascular and systemic features.
CSVD Therapeutics supports a careful translational path: define the cellular phenotype, test candidate interventions, and build the evidence needed for credible therapeutic development.
Project leadership
Dr Klaudia Kocsy leads the COL4A1 therapeutic development space.
Dr Kocsy brings vascular therapeutic development expertise to the COL4A1 project, focusing on disease mechanisms, experimental model strategy, and the careful testing of candidate interventions for COL4A1-related vascular pathology.
Her work connects the biological consequences of COL4A1 mutation with practical therapeutic questions: which disease readouts matter, which interventions are worth testing, and what evidence is needed before a therapeutic concept can move toward translation.
People and projects
A growing COL4A1 research space.
Dr Klaudia Kocsy leads this project, connecting vascular disease biology with therapeutic testing and experimental strategy for COL4A1-related disorders.
Kieran PJ Cawley
Contributed to COL4A1 correction work.
Zuzanna Sokolowska
Tested drug repurposing approaches in COL4A1 disorders.
Dragos Mihai Margarint
Co-tested COL4A1 correction strategies using CRISPR editing.
Luke F Bolger
Tested drug repurposing approaches in COL4A1 disorders.
