-
Şirket
-
Ürünler
-
Hizmetler
-
Akademi
-
Kariyer
Nisan 2023 Bilimsel Takvimi
Hemofili hastalarında gen tedavisi
Hemofili hastalarında gen tedavisine verilen yanıtı izlerken hangi testi kullanırsınız? Tek aşamalı APTT bazlı pıhtılaşma testi mi, kromojenik test mi yoksa başka bir test tipi mi kullanırsınız?
Gen tedavisi gören hemofili A hastalarında kanama riskini izlerken hangi testi kullanırsınız?
Tek aşamalı APTT bazlı (OSAPTT) faktör VIII testi
Kromojenik faktör VIII testi (CFA)
Her iki test – bir tek aşamalı APTT bazlı test ve bir kromonojenik faktör VIII testi – de kullanılır, çünkü her ikisi de kanama fenotipiyle olumlu korelasyon gösterir
Congratulations!
That's the correct answer!
Sorry! That´s not completely correct!
Please try again
Sorry! That's not the correct answer!
Please try again
Notice
Please select at least one answer
Referanslar
[1] Plug I et al. (2006): Mortality and causes of death in patients with haemophilia, 1992–2001. A prospective cohort study. J Thromb Haemost. 4: 510–6.
[2] Tagliaferri A et al. (2010): Mortality and causes of death in Italian persons with haemophilia, 1990–2007. Haemophilia. 16: 437–46.
[3] Mejia-Carvajal C, Czapek EE, Valentino LA. (2006): Life expectancy in hemophilia outcome. J Thromb Haemost. 4: 507–9.
[4] Stephens CJ, Lauron EJ, Kashentseva E, Lu ZH, Yokoyama WM, Curiel DT. (2019): Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9. Journal of Controlled Release. Volume 298, 128–41.
[5] Nataša Savić, Gerald Schwank. (2016): Advances in therapeutic CRISPR/Cas9 genome editing. Translational Research. Volume 168, 15–21.
[6] Batty P, Lillicrap D. (2021): Hemophilia Gene Therapy: Approaching the First Licensed Product. HemaSphere. 5(3), e540.
[7] Ohmori T. (2020): Advances in gene therapy for hemophilia: basics, current status, and future perspectives. International Journal of Hematology. 111: 31–41.
[8] Wu Z, Asokan A, Samulski RJ. (2006): Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy. Molecular Therapy. 14(3): 316–27.
[9] Calcedo R et al. (2009): Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis. 199: 381–90.
[10] Boutin S et al. (2010): Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther. 21: 704–12.
[11] Verdera HC, Kuranda K, Mingozzi F. (2020): AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer. Mol Ther. 28: 723–46.
[12] Majowicz A, Nijmeijer B, Lampen MH et al. (2019): Therapeutic hFIX activity achieved after single AAV5-hFIX treatment in hemophilia B patients and NHPs with pre-existing anti-AAV5 NABs. Mol Ther Methods Clin Dev. 14: 27–36.
[13] Leborgne C et al. (2020): IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies. Nat Med. 26: 1096–1101.
[14] Cantore A, Naldini L. (2021): WFH State-of-the-art paper 2020: In vivo lentiviral vector gene therapy for haemophilia. Haemophilia. 27(Suppl. 3): 122–5.
[15] Rangarajan S et al. (2017): AAV5-factor VIII gene transfer in severe hemophilia A. N Engl J Med. 377: 2519–30.
[16] Nathwani AC, Tuddenham EG, Rangarajan S et al. (2011): Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 365: 2357–65.
[17] Pasi KJ et al. (2020): Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. N Engl J Med. Jan 2; 382(1): 29–40.
[18] EHC news (2022): BioMarin reports a serious adverse event for its phase I/II gene therapy trial for haemophilia A.
[19] Klamroth R. (2022): Gentherapie: Neue Erkenntnisse aus den klinischen Studien. GTH 2022.
[20] George LA et al. (2017): Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med. 377: 2215–27.
[21] Miesbach W et al. (2018): Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood. Mar 1; 131(9): 1022–31.
[22] Pasi K et al. (2021): Hemostatic Response is Maintained for up to 5 Years Following Treatment with Valoctocogene Roxaparvovec, an AAV5-hFVIII-SQ Gene Therapy for Severe Hemophilia A [Abstract]. Res Prac Thromb Haemost. 5 (Suppl 2).
[23] Rosen S et al. (2020): ): Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy. Blood. 126 (22): 2524–34.
[24] Samelson-Jones BJ et al. (2019): Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity. JCI Insight. 4(14): e128683.
