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Pwen esansyèl nan 25yèm Reyinyon Anyèl ASGCT
Livrezon LNP mRNA CRISPR/Cas9 pou repare yon ti sipresyon nan jèn FVIII nan sourit emofili A.
Prezante pa: Chun-Yu Chen, PhD, Fellow-PhD | Immunity & Immunotherapies, Seattle Children's Seattle, Washington, Etazini
Chun-Yu Chen1, Cai Xiaohe1, Carol Miao1,2
1Seattle Children's Research Institute, Seattle, Washington
2Depatman Pedyatri, University of Washington, Seattle, Washington
Kle Done Pwen
- LNP mRNA yo vize tou de epatosit ak LSEC
- Cas9 ak sgRNA LNP yo pèmèt sourit NSG HemA reprann aktivite FVIII nan plasma andojèn.
Divès fòmilasyon luciferase mRNA (Luc) encapsulé nan nanopartikul lipid ki baze sou MC3 (LNP) yo te enjekte nan venn nan sourit yo. Ekspresyon Luciferase te detekte sitou nan fwa, ak ekspresyon ki pi fò yo montre nan fòmilasyon LNP 5. Tach imunostaining fwa te demontre ke LNP ki make Dil ka transfekte avèk siksè tou de epatosit ak selil andotelyal. nan vivo.
Encapsulated Cas9 mRNA ak yon sèl-gid RNA (sgRNA) LNP yo te delivre nan iminofili A (NSG HA) sourit nan venn. Espresyon aPTT te konfime ekspresyon ki estab nan andojèn FVIII pou jiska 4 semèn epi yo te verifye FVIII mitasyon modifye pa sekans ADN ak zouti analiz CRISPR sou entènèt, respektivman.
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