Prospects for gene therapy of haemophilia

Haemophilia : the Official Journal of the World Federation of Hemophilia
A C NathwaniE G D Tuddenham

Abstract

That gene therapy offers the promise of a cure for haemophilia was apparent more than a decade ago. After years of failure, substantial progress in the efficiency of gene transfer technology has recently resulted in impressive success in animal models with haemophilia. However, fears of the risks intrinsic to such therapy have been raised by the fate of two children cured of immune deficiency by gene transfer who have, however, subsequently developed leukaemia as a result of insertional mutagenesis. The purpose of this review is to outline the current status of gene therapy in light of recent successes and tragedies and to consider the prospects for curing haemophilia in the short-to-medium term.

References

Oct 1, 1992·The Journal of Experimental Medicine·R E DonahueR Moen
Sep 22, 1999·Gene Therapy·N ChirmuleJ Wilson
Oct 27, 1999·Proceedings of the National Academy of Sciences of the United States of America·M BurtonL Couto
Mar 20, 2001·Nature Reviews. Genetics·N Somia, I M Verma
Mar 29, 2001·Molecular Therapy : the Journal of the American Society of Gene Therapy·Z Y ChenM A Kay
Jun 9, 2001·The New England Journal of Medicine·D A RothUNKNOWN Factor VIII Transkaryotic Therapy Study Group
Jun 9, 2001·The New England Journal of Medicine·P M Mannucci, E G Tuddenham
Jun 16, 2001·Molecular Therapy : the Journal of the American Society of Gene Therapy·C S SteinP B McCray
Jun 16, 2001·Molecular Therapy : the Journal of the American Society of Gene Therapy·C H MiaoX Ye
Jan 12, 2002·Molecular Therapy : the Journal of the American Society of Gene Therapy·P Seshidhar ReddySheila Connelly
Aug 23, 2002·Proceedings of the National Academy of Sciences of the United States of America·Guang-Ping GaoJames M Wilson
Sep 20, 2002·Journal of Virology·Federico MingozziRoland W Herzog
Jan 17, 2003·The New England Journal of Medicine·Salima Hacein-Bey-AbinaAlain Fischer
Apr 30, 2003·Molecular Therapy : the Journal of the American Society of Gene Therapy·Neeltje A KootstraInder M Verma
Jun 5, 2003·Nature Genetics·Hiroyuki NakaiMark A Kay
Jul 2, 2003·Gene Therapy·J A St George
Jul 9, 2003·Nature Medicine·Hengjun ChaoChristopher E Walsh
Jan 17, 2004·Science·Utpal P DavéNeal G Copeland

❮ Previous
Next ❯

Citations

Oct 29, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Makiko YasudaRobert J Desnick
Jan 20, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Amit C NathwaniAndrew M Davidoff
Jun 2, 2011·Molecular Therapy : the Journal of the American Society of Gene Therapy·Citra N Z MattarJerry K Y Chan
Jun 3, 2005·Nature Reviews. Genetics·Jochen GrawRainer Schwaab
Dec 14, 2011·The New England Journal of Medicine·Amit C NathwaniAndrew M Davidoff
Aug 5, 2006·Current Opinion in Hematology·Katherine P Ponder
Jul 16, 2008·Transplantation·Kohei TatsumiAkira Yoshioka
Feb 7, 2014·International Journal of Hematology·Nishil PatelAmit C Nathwani
Apr 20, 2005·Proceedings of the National Academy of Sciences of the United States of America·Lingfei XuKatherine P Ponder
Apr 13, 2005·Proceedings of the National Academy of Sciences of the United States of America·Mini ThomasAlexander M Klibanov
Jun 5, 2014·BioMed Research International·Agoritsa VarakliotiDimitris Niakas
Mar 11, 2005·Expert Opinion on Emerging Drugs·Guy Young, Louis Aledort
Sep 17, 2013·Ultrasound in Medicine & Biology·Cynthia D AndersonChad B Walton
Apr 20, 2005·Liver Transplantation : Official Publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society·Saiho KoYoshiyuki Nakajima
Jun 11, 2005·Journal of Thrombosis and Haemostasis : JTH·E G D Tuddenham
Jul 27, 2007·The Journal of Gene Medicine·Makiko YasudaRobert J Desnick
Feb 3, 2007·Journal of Thrombosis and Haemostasis : JTH·Q ShiR R Montgomery
Jun 29, 2012·Haemophilia : the Official Journal of the World Federation of Hemophilia·E Tuddenham
Sep 18, 2010·The Journal of Gene Medicine·Inge PetrusThierry VandenDriessche
Jul 17, 2013·Haemophilia : the Official Journal of the World Federation of Hemophilia·K TatsumiT Okano
Apr 26, 2014·Haemophilia : the Official Journal of the World Federation of Hemophilia·K H HighD Lillicrap
Dec 23, 2015·International Journal of Molecular Sciences·Buddhadev LayekJagdish Singh
Dec 21, 2016·The Cochrane Database of Systematic Reviews·Akshay SharmaUlrike M Reiss
Apr 29, 2020·The Cochrane Database of Systematic Reviews·Akshay SharmaUlrike M Reiss
Aug 10, 2020·Current Gene Therapy·Miaojin ZhouDesheng Liang

❮ Previous
Next ❯

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Blood Clotting Disorders

Thrombophilia includes conditions with increased tendency for excessive blood clotting. Blood clotting occurs when the body has insufficient amounts of specialized proteins that make blood clot and stop bleeding. Here is the latest research on blood clotting disorders.

Related Papers

Journal of Thrombosis and Haemostasis : JTH
C Negrier
Circulation Research
Lisa TilemannRoger J Hajjar
© 2022 Meta ULC. All rights reserved