| Project/Area Number |
15H06883
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| Research Category |
Grant-in-Aid for Research Activity Start-up
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| Allocation Type | Single-year Grants |
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| Research Field |
Experimental pathology
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| Research Institution | National Center of Neurology and Psychiatry |
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Principal Investigator |
AOKI YOSHITSUGU 国立研究開発法人国立精神・神経医療研究センター, 神経研究所 遺伝子疾患治療研究部, 室長 (80534172)
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| Project Period (FY) |
2015-08-28 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥2,600,000 (Direct Cost: ¥2,000,000、Indirect Cost: ¥600,000)
Fiscal Year 2016: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2015: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Keywords | デュシェンヌ型筋ジストロフィー / アンチセンス核酸 / モルフォリノ / スカベンジャー受容体 / エクソン 53 スキップ / 薬物送達システム / 筋ジストロフィー / ジストロフィン / 薬物送達 / エクソン・スキップ / クラスA スカベンジャー受容体 / モルフォリノ核酸 / ペプチド付加モルフォリノ核酸 / SR-A欠損マウス / 筋収縮 |
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| Outline of Final Research Achievements |
Duchenne muscular dystrophy (DMD) is an incurable, X-linked progressive muscle degenerative disorder that results from the absence of dystrophin protein.
Here, we study the uptake mechanism of phosphorodiamidate morpholino (PMO), which has been optimized to induce exon skipping in models of DMD. We show that the uptake of PMO is mediated by class A scavenger receptor subtypes (SCARAs) as demonstrated by competitive inhibition in vitro. Also, we newly developed SCARA1 and dystrophin double knockout mice to further confirm the uptake mechanism of PMO in vivo. These results suggest receptor-mediated uptake for PMO.
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