Maltese Maltese
en Englishaf Afrikaanssq Albanianar Arabichy Armenianaz Azerbaijanieu Basquebe Belarusianbg Bulgarianca Catalanzh-CN Chinese (Simplified)zh-TW Chinese (Traditional)hr Croatiancs Czechda Danishnl Dutchet Estoniantl Filipinofi Finnishfr Frenchgl Galicianka Georgiande Germanel Greekht Haitian Creoleiw Hebrewhi Hindihu Hungarianis Icelandicid Indonesianga Irishit Italianja Japaneseko Koreanlv Latvianlt Lithuanianmk Macedonianms Malaymt Malteseno Norwegianfa Persianpl Polishpt Portuguesero Romanianru Russiansr Serbiansk Slovaksl Slovenianes Spanishsw Swahilisv Swedishth Thaitr Turkishuk Ukrainianur Urduvi Vietnamesecy Welshyi Yiddish
David Lillicrap, MD, FRCPC
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

Punti 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).

Punt tad-Dejta 2
Kif jidher f'dan il-graff, il-kelb ittrattat bil-ko-cFVIII-V3 (K2015) żviluppa żewġ episodji ta 'transaminite, elevazzjoni ħafifa ta' wara l-bijopsija f'jum 84, u tieni elevazzjoni akbar f'jum 116, eżatt qabel it-tieni bijopsija. Immaġni bl-ultrasound u bijopsiji tal-fwied użati biex jinvestigaw dawn l-episodji ma wrew l-ebda istopatoloġija sottostanti tal-fwied.

KONTENUT RELATAT

Webinars interattivi
Ippreżentat mill-Prof. Margareth C. Ozelo, MD, PhD ...
Webinars interattivi
Ippreżentat minn Frank WG Leebeek, MD, PhD ...
Webinars interattivi
Ippreżentat minn Lindsey A. George, MD ...
Webinars interattivi
Ippreżentat minn K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPC ...
Webinars interattivi
Ippreżentat minn Guy Young, MD ...
Webinars interattivi
Ippreżentat minn Barbara A. Konkle, MD ...
Webinars interattivi
Ippreżentat minn Johnny Mahlangu, BSc, MBBCh, MMed, FCPath ...
Webinars interattivi
Ippreżentat minn Thierry VandenDriessche, PhD ...
Webinars interattivi
Ippreżentat minn Glenn F. Pierce, MD, PhD ...
Webinars interattivi
Ippreżentat minn Steven W. Pipe, MD ...
Webinars interattivi
Ippreżentat minn David Lillicrap, MD ...
Webinars interattivi
Ippreżentat minn Flora Peyvandi, MD, PhD ...
Webinars interattivi
Lura 'l Fuq
Image

Please enable the javascript to submit this form

dwar

Appoġġjati minn għotjiet edukattivi minn Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, u uniQure, Inc.

SSL essenzjali