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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1964 1
1965 1
1968 10
1969 2
1970 3
1971 2
1972 3
1973 6
1974 4
1975 3
1976 4
1977 2
1979 5
1980 3
1981 9
1982 1
1983 3
1984 3
1985 9
1986 4
1987 6
1988 4
1989 5
1990 3
1991 9
1992 15
1993 15
1994 11
1995 18
1996 9
1997 13
1998 17
1999 17
2000 17
2001 20
2002 15
2003 24
2004 35
2005 12
2006 21
2007 11
2008 25
2009 20
2010 20
2011 35
2012 38
2013 37
2014 44
2015 41
2016 45
2017 37
2018 47
2019 36
2020 25
2021 28
2022 27
2023 38
2024 3

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Search Results

833 results

Results by year

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Page 1
Gene therapy for hemophilia: a review on clinical benefit, limitations, and remaining issues.
Leebeek FWG, Miesbach W. Leebeek FWG, et al. Blood. 2021 Sep 16;138(11):923-931. doi: 10.1182/blood.2019003777. Blood. 2021. PMID: 34232980 Free article. Review.
In the last decade, enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered adeno-associated virus (AAV)-based liver-directed gene therapy in patients with severe …
In the last decade, enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first …
Adeno-Associated Virus Gene Therapy for Hemophilia.
Samelson-Jones BJ, George LA. Samelson-Jones BJ, et al. Annu Rev Med. 2023 Jan 27;74:231-247. doi: 10.1146/annurev-med-043021-033013. Epub 2022 Sep 14. Annu Rev Med. 2023. PMID: 36103998 Free PMC article. Review.
In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy. ...Here we review the clinica …
In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia
Gene therapy for hemophilia.
Nathwani AC. Nathwani AC. Hematology Am Soc Hematol Educ Program. 2022 Dec 9;2022(1):569-578. doi: 10.1182/hematology.2022000388. Hematology Am Soc Hematol Educ Program. 2022. PMID: 36485127 Free PMC article. Review.
Valoctocogene roxaparvovec, the first gene therapy for treatment of hemophilia A, has been granted conditional marketing authorization in Europe. Another approach (etranacogene dezaparvovec, AMT-061) for hemophilia B is also under review by regulators. There …
Valoctocogene roxaparvovec, the first gene therapy for treatment of hemophilia A, has been granted conditional marketing authorizatio …
Haemophilias A and B.
Bolton-Maggs PH, Pasi KJ. Bolton-Maggs PH, et al. Lancet. 2003 May 24;361(9371):1801-9. doi: 10.1016/S0140-6736(03)13405-8. Lancet. 2003. PMID: 12781551 Review.
Although deficiencies of factor VIII (haemophilia A) and factor IX (haemophilia B) are well recognised, von Willebrand's disease is much more common. ...
Although deficiencies of factor VIII (haemophilia A) and factor IX (haemophilia B) are well recognised, von Willebrand's disease is m …
The More Recent History of Hemophilia Treatment.
Franchini M, Mannucci PM. Franchini M, et al. Semin Thromb Hemost. 2022 Nov;48(8):904-910. doi: 10.1055/s-0042-1756188. Epub 2022 Sep 15. Semin Thromb Hemost. 2022. PMID: 36108649 Review.
The treatment of hemophilia has recently reached new landmarks. The traditional clotting factor replacement therapy for hemophilia has been substituted over the last 10 years by novel treatments such as bioengineered factor VIII and IX molecules with extended half-l …
The treatment of hemophilia has recently reached new landmarks. The traditional clotting factor replacement therapy for hemophilia
The past and future of haemophilia: diagnosis, treatments, and its complications.
Peyvandi F, Garagiola I, Young G. Peyvandi F, et al. Lancet. 2016 Jul 9;388(10040):187-97. doi: 10.1016/S0140-6736(15)01123-X. Epub 2016 Feb 18. Lancet. 2016. PMID: 26897598 Review.
Haemophilia A and B are hereditary haemorrhagic disorders characterised by deficiency or dysfunction of coagulation protein factors VIII and IX, respectively. ...Gene therapy has the potential for a definitive cure, and important results have been obtained in haemophilia …
Haemophilia A and B are hereditary haemorrhagic disorders characterised by deficiency or dysfunction of coagulation protein factors V …
Gene Therapy in Hemophilia: Recent Advances.
Rodríguez-Merchán EC, De Pablo-Moreno JA, Liras A. Rodríguez-Merchán EC, et al. Int J Mol Sci. 2021 Jul 17;22(14):7647. doi: 10.3390/ijms22147647. Int J Mol Sci. 2021. PMID: 34299267 Free PMC article. Review.
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. ...In the case of hemophilia A, further research is needed into how to effectively package the large factor VIII gene into the vector; and in the case of hemophilia
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. ...In the case of hemophilia
Hemophilia gene therapy: first, do no harm.
Valentino LA, Kaczmarek R, Pierce GF, Noone D, O'Mahony B, Page D, Rotellini D, Skinner MW. Valentino LA, et al. J Thromb Haemost. 2023 Sep;21(9):2354-2361. doi: 10.1016/j.jtha.2023.06.016. Epub 2023 Jun 21. J Thromb Haemost. 2023. PMID: 37353081 Free article. Review.
Bias on the part of health care professionals and people with hemophilia must be addressed and minimized. Here, we review data leading to the regulatory authorization of valoctocogene roxaparvovec, an adeno-associated virus 5 gene therapy, in Europe to treat hemophilia
Bias on the part of health care professionals and people with hemophilia must be addressed and minimized. Here, we review data leadin …
Etranacogene Dezaparvovec: First Approval.
Heo YA. Heo YA. Drugs. 2023 Mar;83(4):347-352. doi: 10.1007/s40265-023-01845-0. Drugs. 2023. PMID: 36802324 Review.
Etranacogene dezaparvovec (etranacogene dezaparvovec-drlb; Hemgenix()) is an adeno-associated virus vector-based gene therapy being developed by uniQure and CSL Behring for the treatment of haemophilia B. In November 2022, etranacogene dezaparvovec was approved in the USA …
Etranacogene dezaparvovec (etranacogene dezaparvovec-drlb; Hemgenix()) is an adeno-associated virus vector-based gene therapy being develope …
833 results