Arteriovenous malformation, or Arteriovenous vascular malformation (AVM) is a congenic disorder characterized by an abnormal connection between veins and arteries, resulting in hemorrhaging and even death. A lack of good animal models has long been an obstacle for identifying effective drugs for neurological AVM treatment. Describe herein is a mouse model for AVM that includes a viable, postnatal animal with a conditional deletion of the activin receptor-like kinase 1 (Alk1Acvrl1). The Alk1-cKO mouse model can be used to identify genes and gene products that are upregulated in subjects suffering from AVM. For example, it has been discovered Agpt2, IL1β, and TNF-α, are upregulated in Alk1-cKO compared to controls. Pharmaceutical compositions for treatment of AVM are disclosed. Preferred compositions inhibit or decrease expression of angiogenic and pro-inflammatory factors, such as VEFG, Cox-2, Agpt2, IL1β, TNF-α, and matrix metalloproteinases. Methods of determining efficacy of potential AVM therapeutics are also disclosed.
