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Pwen esansyèl Soti nan 28yèm Kongrè Anyèl ESGCT
Siveyans nan sit entegrasyon vektè nan apwòch terapi jenetik in vivo pa likid-byopsy-entegrasyon-sit sekans
D Cesana1, Yon Calabria1, L Rudilosso1, P Gallina1, G Spinozzi1, Yon Magnani2, M Pouzolles3, F Fumagalli1, V Calbi1, M Witzel4, FD Bushman5, A Cantore1, N Taylor3, VS Zimmermann3, C Klein4, Yon Fischer2, M Cavazzana2, E Sis2, A Aiuti1, L Naldini1, E Montini1
1San Raffaele Telethon Enstiti pou Terapi Jèn (HSR-TIGET)
2Laboratwa lenfoematopoèz imen, INSERM, Frans
3Institut de Génétique Moléculaire de Montpellier, CNRS, Frans
4Lopital timoun Dr von Hauner, LMU, Almay
5Depatman Mikwobyoloji, UPenn, USA
Kle Done Pwen
- IS Rekipere Soti nan ADN san selil ki pirifye nan likid kò yo
- Karakterizasyon pwofil entegrasyon Lentivirus (LV) nan chen emofil
ADN san selil (cfDNA) lage nan san an soti nan selil ògàn solid ki an sante, ki anflame oswa ki malad (tankou fwa oswa timis) k ap sibi apoptoz oswa nekwoz. Chanjman jenetik nan cfDNA pirifye, ki gen ladan sit entegrasyon vektè-genomik (IS) soti nan selil transduced, ka detekte pa sekans sit entegrasyon byopsi likid (LiBIS-seq).
Twa chen adilt emofil (O21, M57, ak O59) te resevwa yon sèl piki LV ki eksprime diferan vèsyon nan FIX koagulasyon kanin. Pou chak chen, cfDNA te pirifye nan serom san rekòlte nan 7-8 pwen tan diferan kòmanse soti nan 30 a 532 d apre piki. Grafik sa a montre gwosè popilasyon estime klon selil ki make ki kontribye nan IS yo pataje sou tan nan chen M57 ak O59.
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