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1
Gene therapy for the hemophilias.血友病的基因治疗。
Proc Natl Acad Sci U S A. 1999 Aug 31;96(18):9973-5. doi: 10.1073/pnas.96.18.9973.
2
Gene transfer in the hemophilias: retrospect and prospect.血友病中的基因转移：回顾与展望。
Thromb Res. 1992 Aug 1;67(3):329-38. doi: 10.1016/0049-3848(92)90008-x.
3
Hemophilia clinical gene therapy: brief review.血友病临床基因治疗：简要综述。
Transl Res. 2013 Apr;161(4):307-12. doi: 10.1016/j.trsl.2012.12.016. Epub 2013 Jan 23.
4
Gene therapy of the hemophilias.血友病的基因治疗。
Semin Hematol. 2004 Oct;41(4):287-96. doi: 10.1053/j.seminhematol.2004.07.005.
5
State and prospects of gene therapy for the hemophilias.
Thromb Haemost. 1995 Jul;74(1):266-73.
6
Gene therapy for hemophilia.血友病的基因治疗。
Artif Cells Blood Substit Immobil Biotechnol. 2000 Jan;28(1):1-24. doi: 10.3109/10731190009119782.
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Correction of the coagulation defect in hemophilia A mice through factor VIII expression in skin.通过在皮肤中表达凝血因子VIII纠正甲型血友病小鼠的凝血缺陷。
Blood. 2000 May 1;95(9):2799-805.
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Gene therapy for the hemophilias.
Curr Opin Pediatr. 2002 Feb;14(1):12-6. doi: 10.1097/00008480-200202000-00003.
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Hemophilia as a blueprint for gene therapy.血友病作为基因治疗的蓝本。
Science. 2021 Oct;374(6563):40-41. doi: 10.1126/science.abg0856. Epub 2021 Sep 30.
10
Gene therapy and the hemophilias.基因治疗与血友病
JAMA. 1994 Jan 5;271(1):47-51.

引用本文的文献

1
Transamniotic Delivery of Coagulation Factor VIII mRNA: A Step Toward a Potential Novel Strategy for the Perinatal Management of Hemophilia A.凝血因子VIII mRNA的经羊膜递送：迈向血友病A围产期管理潜在新策略的一步。
FASEB Bioadv. 2025 Sep 3;7(8):e70047. doi: 10.1096/fba.2025-00200. eCollection 2025 Aug.
2
Hemophilia A: An Ideal Disease for Prenatal Therapy.甲型血友病：产前治疗的理想疾病。
Prenat Diagn. 2025 Jun 10. doi: 10.1002/pd.6833.
3
Gene modification therapies for hereditary diseases in the fetus.胎儿遗传性疾病的基因修饰疗法。
Prenat Diagn. 2023 May;43(5):674-686. doi: 10.1002/pd.6347. Epub 2023 Apr 5.
4
Mechanistic Insights into Factor VIII Immune Tolerance Induction via Prenatal Cell Therapy in Hemophilia A.对血友病A中通过产前细胞疗法诱导凝血因子VIII免疫耐受的机制性见解。
Curr Stem Cell Rep. 2019 Dec;5(4):145-161. doi: 10.1007/s40778-019-00165-y. Epub 2019 Nov 20.
5
Flow-Cytometry Platform for Intracellular Detection of FVIII in Blood Cells: A New Tool to Assess Gene Therapy Efficiency for Hemophilia A.用于血细胞内FVIII检测的流式细胞术平台：评估A型血友病基因治疗效率的新工具。
Mol Ther Methods Clin Dev. 2019 Nov 15;17:1-12. doi: 10.1016/j.omtm.2019.11.003. eCollection 2020 Jun 12.
6
Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes.新型腺病毒载体用于B型血友病的新生儿基因治疗，该载体显示病毒基因的渗漏表达减少。
Mol Ther Methods Clin Dev. 2017 Jul 8;6:183-193. doi: 10.1016/j.omtm.2017.07.001. eCollection 2017 Sep 15.
7
In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application.子宫内干细胞移植和基因治疗：原理、历史和临床应用的最新进展。
Mol Ther Methods Clin Dev. 2016 Mar 30;5:16020. doi: 10.1038/mtm.2016.20. eCollection 2016.
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Hemophilia A: an ideal disease to correct in utero.甲型血友病：一种适合在子宫内进行矫正的理想疾病。
Front Pharmacol. 2014 Dec 11;5:276. doi: 10.3389/fphar.2014.00276. eCollection 2014.
9
Non-viral FoxM1 gene delivery to hepatocytes enhances liver repopulation.非病毒载体介导的FoxM1基因导入肝细胞可增强肝脏细胞再生。
Cell Death Dis. 2014 May 22;5(5):e1252. doi: 10.1038/cddis.2014.230.
10
Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene Delivery.以绵羊为细胞和基因递送模型对血友病A进行产前和产后治疗。
J Genet Syndr Gene Ther. 2012 May 25;S1. doi: 10.4172/2157-7412.S1-011.

本文引用的文献

1
Plasma thromboplastin component (PTC) deficiency; a new disease resembling hemophilia.血浆凝血活酶成分（PTC）缺乏症；一种类似血友病的新疾病。
Proc Soc Exp Biol Med. 1952 Apr;79(4):692-4. doi: 10.3181/00379727-79-19488.
2
Christmas disease: a condition previously mistaken for haemophilia.克里斯马斯病：一种曾被误诊为血友病的病症。
Br Med J. 1952 Dec 27;2(4799):1378-82. doi: 10.1136/bmj.2.4799.1378.
3
Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice.人凝血因子VIII在VIII因子缺陷小鼠体内经逆转录病毒基因转移后的长期表达及甲型血友病的纠正
Proc Natl Acad Sci U S A. 1999 Aug 31;96(18):10379-84. doi: 10.1073/pnas.96.18.10379.
4
Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors.使用肝脏特异性启动子可降低腺病毒载体中转基因的免疫反应。
Hum Gene Ther. 1999 Jul 20;10(11):1773-81. doi: 10.1089/10430349950017455.
5
Implication of interfering antibody formation and apoptosis as two different mechanisms leading to variable duration of adenovirus-mediated transgene expression in immune-competent mice.在免疫健全的小鼠中，干扰抗体形成和细胞凋亡作为导致腺病毒介导的转基因表达持续时间可变的两种不同机制的影响。
J Virol. 1999 Jun;73(6):4755-66. doi: 10.1128/JVI.73.6.4755-4766.1999.
6
Sustained correction of bleeding disorder in hemophilia B mice by gene therapy.基因疗法对B型血友病小鼠出血性疾病的持续纠正。
Proc Natl Acad Sci U S A. 1999 Mar 30;96(7):3906-10. doi: 10.1073/pnas.96.7.3906.
7
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors.使用重组腺相关病毒载体在犬类和鼠类模型中纠正血友病B。
Nat Med. 1999 Jan;5(1):64-70. doi: 10.1038/4751.
8
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector.腺相关病毒载体介导的凝血因子IX基因转移对犬血友病B的长期纠正。
Nat Med. 1999 Jan;5(1):56-63. doi: 10.1038/4743.
9
Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice.肝细胞生长因子可在体内诱导肝细胞增殖，并有利于在小鼠中进行高效的逆转录病毒介导的基因转移。
Hepatology. 1998 Sep;28(3):707-16. doi: 10.1002/hep.510280317.
10
Targeted inactivation of the coagulation factor IX gene causes hemophilia B in mice.
Blood. 1998 Jul 1;92(1):168-74.

Gene therapy for the hemophilias.

作者信息

Kay M A, High K

出版信息

Proc Natl Acad Sci U S A. 1999 Aug 31;96(18):9973-5. doi: 10.1073/pnas.96.18.9973.

DOI:10.1073/pnas.96.18.9973
PMID:10468539
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC33717/
Abstract
摘要