Worldwide use of factor IX Padua for hemophilia B gene therapy.
作者信息
Samelson-Jones Benjamin J
机构信息
Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, 5028 Colket Translational Research Center 3501 Civic Center Blvd., Philadelphia, PA 19104, USA; Department of Pediatrics, Division of Hematology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
出版信息
Mol Ther. 2022 Jul 6;30(7):2394-2396. doi: 10.1016/j.ymthe.2022.06.002. Epub 2022 Jun 14.
Abstract
摘要
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本文引用的文献
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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.携带高活性帕多瓦因子 IX 的工程化、肝脏靶向腺相关病毒载体在接受预防性糖皮质激素治疗的乙型血友病患者中的安全性和活性：一项单中心、单臂、1 期、初步试验。
Lancet Haematol. 2022 Jul;9(7):e504-e513. doi: 10.1016/S2352-3026(22)00113-2. Epub 2022 May 19.
2
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette.使用工程化腺相关病毒3型衣壳和肝脏优化表达盒将凝血因子IX基因转移至非人灵长类动物。
Mol Ther Methods Clin Dev. 2021 Aug 26;23:98-107. doi: 10.1016/j.omtm.2021.08.001. eCollection 2021 Dec 10.
3
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua.在使用超功能变异因子 IX-Padua 进行肝脏基因治疗的情况下，因子 IX 测定结果存在差异。
J Thromb Haemost. 2021 May;19(5):1212-1218. doi: 10.1111/jth.15281. Epub 2021 Mar 28.
4
Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.依特兰基因疗法（AMT-061 二期临床试验）：乙型血友病患者体内正常/接近正常的因子 IX 活性和出血停止。
Blood Adv. 2019 Nov 12;3(21):3241-3247. doi: 10.1182/bloodadvances.2019000811.
5
Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity.因子 IX Padua（R338L）的过度活跃依赖于因子 VIIIa 辅因子活性。
JCI Insight. 2019 Jun 20;5(14):128683. doi: 10.1172/jci.insight.128683.
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Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model.FIX-Padua 骨骼肌基因治疗纠正血友病 B 表型，抑制物倾向犬模型。
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.采用高特异性活性因子IX变体的B型血友病基因疗法。
N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.
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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.在血友病B犬和小鼠中，腺相关病毒介导的肝脏表达FIX-Padua可预防和消除FIX抑制剂，且不会增加血栓形成倾向。
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Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice.高功能凝血因子 IX 提高血友病小鼠基因治疗的疗效。
Blood. 2012 Nov 29;120(23):4517-20. doi: 10.1182/blood-2012-05-432591. Epub 2012 Oct 4.
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The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.用 AAV 肌肉基因疗法治疗的血友病 B 犬中 FIX Padua（R338L）的疗效和免疫原性风险。
Blood. 2012 Nov 29;120(23):4521-3. doi: 10.1182/blood-2012-06-440123. Epub 2012 Aug 23.
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