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Punti ewlenin mill-Kungress ISTH 2021
Investigazzjoni ta 'Riżultati Bikrija Wara Terapija tal-Ġene Virali assoċjata ma' Adeno f'Mudell ta 'Emofilja tal-Klieb
P. Batty1, B. Yates2, D. Hurlbut3, S. Siso-Llonch2, A. Menard3, H. Akeefe2, T. Christianson2, P. Colosi2, L. Harpell1, S. Liu2, A. Mo1, A. Pender1, G. Veres2, C. Vitelli2, A. Winterborn4, S. Bunting2, S. Fong2, D. Lillicrap1
1Dipartiment tal-Patoloġija u l-Mediċina Molekulari, Queen's University, Kingston, il-Kanada
2BioMarin Pharmaceutical, Novato, l-Istati Uniti
3Kingston Health Sciences, Kingston, il-Kanada
4Servizzi ta 'Kura tal-Annimali, Queen's University, Kingston, il-Kanada
Punti Ewlenin tad-Dejta
Emofilja Mudell ta 'kelb intuża biex jevalwa bidliet li jaffettwaw il-fwied u fatturi li jaffettwaw l-espressjoni ta' FVIII wara terapija tal-ġene AAV. Ħames klieb irġiel ġew ikkurati b'infużjoni waħda ta 'AAV5-HLP-cFVIII f'dożi ta' 6.8e13-2e14 vector genomes / kg. L-espressjoni FVIII dehret għat-2 klieb ikkurati b'kostrotti ottimizzati għall-codon (JA2013 u K2015), li kienet sostnuta f'sitt xhur (co-cFVIII-SQ = 6.8% u co-cFVIII-V3 = 4.3%) bin-normalizzazzjoni tat-totalità ħin ta 'tagħqid tad-demm (WBCT).
KONTENUT RELATAT
