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Itilize afèz yo pou yo retire Antibò AAV netralize nan pasyan yo ekskli de Terapi jèn
Pwen esansyèl ki soti nan ASGCT 23rd Reyinyon Anyèl la

Itilize afèz yo pou yo retire Antibò AAV netralize nan pasyan yo ekskli de Terapi jèn

Jonatan H. Foley1Erald Shehu1, Allison Dane1, Rose Sheridan1, Rebeka Alade1, Thorold Guy1, Jenny McIntosh2, Hattie Ollerton1Sophie Williams ...1Romuald Corbau1, Helen Jones2, Natan Davies2, Andre Davenport2, David Briggs3Amit Nathwani2

1Freeline, Stevenage, Wayòm Ini

2University College London, London, Wayòm Ini

3NHS san ak transplantasyon, Birmingham, Wayòm Ini

Kle Done Pwen

Jiska 70% nan pasyan ki gen emofili ka eksprime antikò ki anpeche tretman ak vektè AAV pou terapi jèn. Etid sa a montre ke yo kapab itilize plasmapherèz doub filtraj (DFPP) pou redwi titav AAVS3 netralize antikò (NAb) pou aliyen ak kritè elijibilite jijman.

Rezilta ki sòti nan pasyan ki gen oswa san sik DFPP youn apre lòt lè l sèvi avèk klere luciferase transdiksyon anpèchman tès (BL-TIA). Sik konsekitif yo te pi efikas pase sik ki pa youn apre lòt nan diminye tit tit. Analiz retou annaryè prevwa ke 5 sik ta diminye kòmanse AAVS3 NAb tit pa 94%, ki ta pèmèt tretman 60% nan pasyan yo.

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Sipòte pa sibvansyon edikasyon bay Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, ak uniQure, Inc.

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