为甲型血友病基因治疗铺平道路。 - Suppr

Prepare the Way for Hemophilia A Gene Therapy.

作者信息

Thornburg Courtney D

机构信息

From the Hemophilia and Thrombosis Treatment Center, Rady Children's Hospital San Diego, San Diego, and the Department of Pediatrics, University of California San Diego Health Sciences, La Jolla.

出版信息

N Engl J Med. 2022 Mar 17;386(11):1081-1082. doi: 10.1056/NEJMe2200878.

DOI:10.1056/NEJMe2200878

PMID:35294817

Abstract

摘要

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Prepare the Way for Hemophilia A Gene Therapy.

N Engl J Med. 2022 Mar 17;386(11):1081-1082. doi: 10.1056/NEJMe2200878.

[Recent advance in hemophilia therapy (2): gene and cell therapy for hemophilia].

Rinsho Ketsueki. 2014 Aug;55(8):899-907.

Gene therapy for hemophilia: the clot thickens.

Hum Gene Ther. 2014 Nov;25(11):915-22. doi: 10.1089/hum.2014.2541.

[Hemophilia].

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[Gene therapy of hemophilia].

Internist (Berl). 2001 Oct;42(10):1366-8, 1371-3. doi: 10.1007/s001080170058.

Gene Therapy for Hemophilia A.

N Engl J Med. 2022 Jun 9;386(23):2247. doi: 10.1056/NEJMc2204934.

Gene Therapy for Hemophilia A. Reply.

N Engl J Med. 2022 Jun 9;386(23):2247. doi: 10.1056/NEJMc2204934.

Long-Term Effects of Hemophilia B Gene Therapy.

N Engl J Med. 2023 May 18;388(20):1918. doi: 10.1056/NEJMc2304262.

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Long-Term Effects of Hemophilia B Gene Therapy. Reply.

N Engl J Med. 2023 May 18;388(20):1918-1919. doi: 10.1056/NEJMc2304262.

引用本文的文献

Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus.

Front Med (Lausanne). 2023 Nov 10;10:1256919. doi: 10.3389/fmed.2023.1256919. eCollection 2023.

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.

Expert Rev Hematol. 2023 Mar;16(sup1):19-37. doi: 10.1080/17474086.2023.2171981.

How to translate and implement the current science of gene therapy into haemophilia care?

Ther Adv Hematol. 2023 Jan 12;14:20406207221145627. doi: 10.1177/20406207221145627. eCollection 2023.

Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.

Haemophilia. 2022 Sep;28(5):760-768. doi: 10.1111/hae.14588. Epub 2022 Jun 14.

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相似文献

Prepare the Way for Hemophilia A Gene Therapy.

N Engl J Med. 2022 Mar 17;386(11):1081-1082. doi: 10.1056/NEJMe2200878.

[Recent advance in hemophilia therapy (2): gene and cell therapy for hemophilia].

Rinsho Ketsueki. 2014 Aug;55(8):899-907.

Gene therapy for hemophilia: the clot thickens.

Hum Gene Ther. 2014 Nov;25(11):915-22. doi: 10.1089/hum.2014.2541.

[Hemophilia].

Rinsho Ketsueki. 2013 Feb;54(2):189-97.

[Gene therapy of hemophilia].

Internist (Berl). 2001 Oct;42(10):1366-8, 1371-3. doi: 10.1007/s001080170058.

Gene Therapy for Hemophilia A.

N Engl J Med. 2022 Jun 9;386(23):2247. doi: 10.1056/NEJMc2204934.

Gene Therapy for Hemophilia A. Reply.

N Engl J Med. 2022 Jun 9;386(23):2247. doi: 10.1056/NEJMc2204934.

Long-Term Effects of Hemophilia B Gene Therapy.

N Engl J Med. 2023 May 18;388(20):1918. doi: 10.1056/NEJMc2304262.

EU gets first hemophilia B gene therapy.

Nat Biotechnol. 2023 Apr;41(4):438. doi: 10.1038/s41587-023-01760-5.

Long-Term Effects of Hemophilia B Gene Therapy. Reply.

N Engl J Med. 2023 May 18;388(20):1918-1919. doi: 10.1056/NEJMc2304262.

引用本文的文献

Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus.

Front Med (Lausanne). 2023 Nov 10;10:1256919. doi: 10.3389/fmed.2023.1256919. eCollection 2023.

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.

Expert Rev Hematol. 2023 Mar;16(sup1):19-37. doi: 10.1080/17474086.2023.2171981.

How to translate and implement the current science of gene therapy into haemophilia care?

Ther Adv Hematol. 2023 Jan 12;14:20406207221145627. doi: 10.1177/20406207221145627. eCollection 2023.

Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.

Haemophilia. 2022 Sep;28(5):760-768. doi: 10.1111/hae.14588. Epub 2022 Jun 14.

Prepare the Way for Hemophilia A Gene Therapy.

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Prepare the Way for Hemophilia A Gene Therapy.

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