An Endogenous Anti-angiogenic Protein (EAP) and its Derivatives for Treatment of Cerebral Cavernous Malformations (CCM)
- 技術優勢
- Prior studies have shown that administration of recombinant EAP and/or its analogues are feasible in animal models and one Phase I testing in humans resulted in no serious adverse reactions, demonstrating that EAP-based therapies are feasible and that ultimately small molecule orally-available agents that mimic EAP may be developed to treat these patients.
- 技術應用
- Recombinant EAP, its analogues, or derivatives offer potential therapies to prevent CCM lesion development and progression.
- 詳細技術說明
- EAP is involved in the maintenance of vascular structure and homeostasis. Researchers at UC San Diego found that upon mutation of CCM1/KRIT1 gene, EAP expression is suppressed. Replacement of EAP is a novel potential therapy for CCM disease. Moreover, recombinant fragments of EAP have been shown to be therapeutic in a mouse CCM model of the disease.
- *Abstract
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Cerebral cavernous malformation (CCM) is a neurovascular disease that causes epilepsy and stroke for which there is no medical therapy. It has a prevalence of 5 per thousand in western populations and occurs in familial forms as a consequence of mutations in 3 CCM genes: CCM1/KRIT1, CCM2, CCM3/PCDC10 resulting in the formation of CCMs; mutations in the CCM1/KRIT1 gene account for 40% of the inherited cases. Once identified, CCM patients have a lifetime risk of CCM development and progression with increasing risk of stroke, epilepsy, or neurological impairment.
- *IP Issue Date
- Oct 19, 2017
- *Principal Investigation
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Name: Mark Ginsberg
Department:
Name: Miguel Lopez-Ramirez
Department:
- 其他
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Intellectual Property Info
A provisional patent has been submitted.
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Tech ID/UC Case
27312/2016-214-0
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2016-214-0
- 國家/地區
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