脳の洞穴性不形成の治療のために再利用された薬物を特定するための戦略

Christopher C Gibson1, Weiquan Zhu1, Chadwick T Davis1

  • 1From the Program in Molecular Medicine (C.C.G., W.Z., C.T.D., J.A.B.-K., A.C.C., J.L., A.H.G., K.R.T., K.J.W., D.Y.L.), Department of Bioengineering (C.C.G., Y.-T.E.S.), Department of Medicine (C.C.G., W.Z., K.R.T., D.Y.L.), Department of Human Genetics (C.T.D.), Department of Oncological Sciences (A.C.C., D.Y.L.), Division of Geriatrics, Department of Medicine (A.E.W., A.J.D., L.A.L.), Division of Nephrology and Hypertension, Department of Medicine (Y.-T.E.S.), Department of Pathology (A.H.G.), Division of Cardiology, and Department of Medicine (K.J.W., D.Y.L.), University of Utah, Salt Lake City, UT; Recursion Pharmaceuticals, LLC, Salt Lake City, UT (C.C.G., D.Y.L.); CCM Italia, Department of Clinical and Biological Sciences, University of Torino, Orbassano, Torino, Italy (L.G., S.F.R.); CCM Italia, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, Italy (S.D.M.); Geriatrics Research Education and Clinical Center, Veteran's Affairs Medical Center, Salt Lake City, UT (A.J.D., L.A.L.); The Key Laboratory for Human Disease Gene Study of Sichuan Province, Institute of Laboratory Medicine, Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital, Chengdu, Sichuan, China (D.Y.L.); and Cardiology Section, VA Salt Lake City Health Care System, Salt Lake City, UT (K.J.W., O.Y.L.).

Circulation
|December 10, 2014
PubMed
まとめ

研究者らは,既存の2つの薬物,ビタミンD3とテンポルを,出血性脳卒中症候群である脳洞性形 (CCM) の潜在的な治療法として特定した. これらの化合物は,臨床前モデルの疾患負担を大幅に軽減し,新しい治療法へのより速い経路を提供しました.