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Quoted phrase not found in phrase index: "Increased factor IX activity"
Page 1
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Pipe SW, et al. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
BACKGROUND: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against blee …
BACKGROUND: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent …
Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Chowdary P, et al. N Engl J Med. 2022 Jul 21;387(3):237-247. doi: 10.1056/NEJMoa2119913. N Engl J Med. 2022. PMID: 35857660 Free article. Clinical Trial.
After receiving the infusion, all the patients had dose-dependent increases in factor IX levels. At a median follow-up of 27.2 months (range, 19.1 to 42.4), sustained factor IX activity was observed in all the patients except one, …
After receiving the infusion, all the patients had dose-dependent increases in factor IX levels. At a median follow-up …
Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial.
Xue F, Li H, Wu X, Liu W, Zhang F, Tang D, Chen Y, Wang W, Chi Y, Zheng J, Du Z, Jiang W, Zhong C, Wei J, Zhu P, Fu R, Liu X, Chen L, Pei X, Sun J, Cheng T, Yang R, Xiao X, Zhang L. Xue F, et al. Lancet Haematol. 2022 Jul;9(7):e504-e513. doi: 10.1016/S2352-3026(22)00113-2. Epub 2022 May 19. Lancet Haematol. 2022. PMID: 35598604 Clinical Trial.
BACKGROUND: A novel, engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX (FIX) protein (BBM-H901) has been developed and is promising for haemophilia B gene therapy. We aimed to explore its safety and activity in …
BACKGROUND: A novel, engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX (FIX) pro …
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. Nathwani AC, et al. N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10. N Engl J Med. 2011. PMID: 22149959 Free PMC article. Clinical Trial.
METHODS: We infused a single dose of a serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a codon-optimized human factor IX (FIX) transgene (scAAV2/8-LP1-hFIXco) in a peripheral vein in six patients with severe hemophilia …
METHODS: We infused a single dose of a serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a codo …
Increased bone resorption in hemophilia.
Rodriguez-Merchan EC, Valentino LA. Rodriguez-Merchan EC, et al. Blood Rev. 2019 Jan;33:6-10. doi: 10.1016/j.blre.2018.05.002. Epub 2018 May 25. Blood Rev. 2019. PMID: 29857920 Review.
A narrative review of the English literature up to April 2018 was performed. The available evidence demonstrates an increased rate of bone resorption and an excess of osteoporosis among patients with hemophilia. ...The role of confounders such as lack of activity an …
A narrative review of the English literature up to April 2018 was performed. The available evidence demonstrates an increased rate of …
Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants.
Samelson-Jones BJ, Finn JD, Raffini LJ, Merricks EP, Camire RM, Nichols TC, Arruda VR. Samelson-Jones BJ, et al. Blood Adv. 2021 Mar 9;5(5):1324-1332. doi: 10.1182/bloodadvances.2019000405. Blood Adv. 2021. PMID: 33656538 Free PMC article.
The high-specific-activity factor IX (FIX) variant Padua (R338L) is the most promising transgene for hemophilia B (HB) gene therapy. ...Notably, we observe that hFIX-R338L is the most active variant and cFIX-R338L is sevenfold higher than wild-t …
The high-specific-activity factor IX (FIX) variant Padua (R338L) is the most promising transgene for hemophilia B (HB) …
Considerations on activity assay discrepancies in factor VIII and factor IX products.
Ovanesov MV, Jackson JW, Golding B, Lee TK. Ovanesov MV, et al. J Thromb Haemost. 2021 Sep;19(9):2102-2111. doi: 10.1111/jth.15425. Epub 2021 Jul 20. J Thromb Haemost. 2021. PMID: 34145730 Free article. Review.
New modified coagulation factor VIII (FVIII) and factor IX (FIX) products have been designed to improve the treatment of individuals with hemophilia A and B by increasing the interval between dosing. ...The assignment of potencies for these products ca …
New modified coagulation factor VIII (FVIII) and factor IX (FIX) products have been designed to improve the treatment o …
Improved activity and expression of recombinant human factor IX by propeptide engineering.
Vatandoost J, Bos MHA. Vatandoost J, et al. Daru. 2019 Dec;27(2):653-660. doi: 10.1007/s40199-019-00299-8. Epub 2019 Oct 21. Daru. 2019. PMID: 31637661 Free PMC article.
PURPOSE: The main therapeutic strategy for Hemophilia B patients involves the administration of recombinant coagulation factors IX (rFIX). Although there are various approaches to increasing the activity of rFIX, targeted protein engineering of specific resid …
PURPOSE: The main therapeutic strategy for Hemophilia B patients involves the administration of recombinant coagulation factors IX (r …
Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity.
Samelson-Jones BJ, Finn JD, George LA, Camire RM, Arruda VR. Samelson-Jones BJ, et al. JCI Insight. 2019 Jun 20;5(14):e128683. doi: 10.1172/jci.insight.128683. JCI Insight. 2019. PMID: 31219805 Free PMC article.
Recently, this obstacle has been partially circumvented by the use of a hyperactive factor IX (FIX) variant, R338L (Padua), which has an eightfold increased specific activity compared to FIX-WT. ...We observed that the high-specific-activity …
Recently, this obstacle has been partially circumvented by the use of a hyperactive factor IX (FIX) variant, R338L (Padua), wh …
Activity measurements of dalcinonacog alfa.
Williams SC, Gray E. Williams SC, et al. Haemophilia. 2020 Mar;26(2):346-353. doi: 10.1111/hae.13949. Epub 2020 Mar 6. Haemophilia. 2020. PMID: 32142203 Free PMC article.
No statistically valid results were obtained when testing dalcinonacog alfa using either chromogenic kit. Increasing the incubation time with the activation reagent in both chromogenic kits resulted in valid assays and increased the potency to become more in …
No statistically valid results were obtained when testing dalcinonacog alfa using either chromogenic kit. Increasing the incubation t …
401 results