Related Experiment Video
Updated: Jul 26, 2025
In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium
Published on: May 6, 2018
Focal Anticoagulation by Somatic Gene Transfer: Towards Preventing Cardioembolic Stroke
Megha Kadian1, Cindy Y Kok2, Dhanya Ravindran2
1The Centre for Heart Research, The Westmead Institute for Medical Research, Sydney, NSW, Australia; The University of Sydney, Sydney, NSW, Australia; Faculty of Medicine, The University of Queensland, St Lucia, Qld, Australia.
Insights
Cardioembolic stroke (CS) is a major cause of ischemic stroke (IS). Gene therapy offers a novel approach to target thrombosis causes, addressing limitations of current anticoagulation treatments for high-risk patients.
Area of Science:
- Neurology
- Cardiology
- Genetics
Background:
- Cardioembolic stroke (CS), often originating from the left atrial appendage (LAA), is a leading cause of ischemic stroke (IS).
- Current treatments like systemic anticoagulation are not personalized and have contraindications, leaving high-risk patients vulnerable.
- Atrial appendage occlusion devices mitigate LAA-thrombus risk but are costly, invasive, and don't address underlying causes.
Purpose of the Study:
- To explore the potential of viral vector-based gene therapy for treating cardioembolic stroke (CS).
- To address the gap in research regarding adeno-associated virus (AAV) gene therapy for thrombotic disorders like CS.
- To investigate gene therapy's ability to target molecular pathways promoting thrombosis in CS.
Main Methods:
- Literature review on current CS treatments and gene therapy applications in haemostatic disorders.
- Analysis of the potential of adeno-associated virus (AAV) vectors for targeted gene delivery.
- Exploration of gene therapy's capacity to address the molecular etiology of CS.
Main Results:
- Systemic anticoagulation and device-based therapies have limitations for CS management.
- Adeno-associated virus (AAV) gene therapy has shown success in other haemostatic disorders like haemophilia.
- Gene therapy presents a novel strategy for localized, targeted treatment of CS by addressing thrombosis mechanisms.
Conclusions:
- Gene therapy holds promise as a personalized and targeted approach for cardioembolic stroke (CS).
- Further research into AAV-based gene therapy for CS is warranted to address unmet clinical needs.
- Targeting the molecular basis of thrombosis offers a potential paradigm shift in CS treatment.
Abstract:
Cardioembolic stroke (CS) has emerged as a leading cause of ischaemic stroke (IS); distinguished by thrombi embolising to the brain from cardiac origins; most often from the left atrial appendage (LAA). Contemporary therapeutic options are largely dependent on systemic anticoagulation as a blanket preventative strategy, yet this does not represent a nuanced or personalised solution. Contraindications to systemic anticoagulation create significant unmedicated and high-risk cohorts, leaving these patients at risk of significant morbidity and mortality. Atrial appendage occlusion devices are increasingly used to mitigate stroke risk from thrombi emerging from the LAA in patients ineligible for oral anticoagulants (OACs). Their use, however, is not without risk or significant cost, and does not address the underlying aetiology of thrombosis and CS. Viral vector-based gene therapy has emerged as a novel strategy to target a spectrum of haemostatic disorders, achieving success through the adeno-associated virus (AAV) based therapy of haemophilia. Yet, thrombotic disorders, such as CS, have had limited exploration within the realm of AAV gene therapy approaches-presenting a gap in the literature and an opportunity for further research. Gene therapy has the potential to directly address the cause of CS by localised targeting of the molecular remodelling that serves to promote thrombosis.
More Related Videos
Related Concept Videos
01:30Anticoagulant Drugs: Low-Molecular-Weight Heparins
01:29Venous Thrombosis III: Interprofessional Care
01:18Anticoagulant Drugs: Vitamin K Antagonists and Direct Oral Anticoagulants
Warfarin, a prominent vitamin K antagonist family member, exerts its effect by inhibiting the enzyme VKORC1 (vitamin K epoxide reductase complex 1). By hindering this enzyme, warfarin...
00:59Gene Therapy
01:16Clot Retraction and Fibrinolysis