Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
J McIntosh, PJ Lenting, C Rosales… - Blood, The Journal …, 2013 - ashpublications.org
Recombinant adeno-associated virus (rAAV) vectors encoding human factor VIII (hFVIII)
were systematically evaluated for hemophilia A (HA) gene therapy. A 5.7-kb rAAV …
were systematically evaluated for hemophilia A (HA) gene therapy. A 5.7-kb rAAV …
Activity of transgene-produced B-domain–deleted factor VIII in human plasma following AAV5 gene therapy
S Rosen, S Tiefenbacher, M Robinson… - Blood, The Journal …, 2020 - ashpublications.org
Abstract Adeno-associated virus (AAV)-based gene therapies can restore endogenous
factor VIII (FVIII) expression in hemophilia A (HA). AAV vectors typically use a B-domain …
factor VIII (FVIII) expression in hemophilia A (HA). AAV vectors typically use a B-domain …
Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII
CD Scallan, T Liu, AE Parker, SL Patarroyo-White… - Blood, 2003 - ashpublications.org
Using separate adeno-associated viral 2 (AAV2) vectors to deliver the heavy and light
chains of factor VIII (FVIII) we have overcome the packaging limitations of AAV, achieving …
chains of factor VIII (FVIII) we have overcome the packaging limitations of AAV, achieving …
Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
MKL Chuah, G Schiedner, L Thorrez… - Blood, The Journal …, 2003 - ashpublications.org
High-capacity adenoviral (HC-Ad) vectors expressing B-domain–deleted human or canine
factor VIII from different liver-specific promoters were evaluated for gene therapy of …
factor VIII from different liver-specific promoters were evaluated for gene therapy of …
Sustained expression of human factor VIII in mice using a parvovirus-based vector
H Chao, L Mao, AT Bruce… - Blood, The Journal of the …, 2000 - ashpublications.org
Persistent therapeutic levels of human factor VIII (hFVIII) would signify a major advance in
the treatment of hemophilia A. Here we report sustained expression of hFVIII in …
the treatment of hemophilia A. Here we report sustained expression of hFVIII in …
Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
C Balagué, J Zhou, Y Dai, R Alemany… - Blood, The Journal …, 2000 - ashpublications.org
The successful prophylactic treatment of hemophilia A by frequent infusions of plasma
concentrates or recombinant factor VIII (hFVIII) indicates that gene therapy may be a …
concentrates or recombinant factor VIII (hFVIII) indicates that gene therapy may be a …
Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe …
BD Brown, CX Shi, S Powell, D Hurlbut, FL Graham… - Blood, 2004 - ashpublications.org
Two helper-dependent (HD) adenoviral vectors encoding a canine factor VIII B-domain–
deleted transgene (cFVIII) were constructed and evaluated in 4 hemophilia A dogs. One …
deleted transgene (cFVIII) were constructed and evaluated in 4 hemophilia A dogs. One …
[HTML][HTML] Multiyear factor VIII expression after AAV gene transfer for hemophilia A
LA George, PE Monahan, ME Eyster… - … England Journal of …, 2021 - Mass Medical Soc
Background The goal of gene therapy for patients with hemophilia A is to safely impart long-
term stable factor VIII expression that predictably ameliorates bleeding with the use of the …
term stable factor VIII expression that predictably ameliorates bleeding with the use of the …
Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
M Burton, H Nakai, P Colosi… - Proceedings of the …, 1999 - National Acad Sciences
We are interested in using recombinant adeno-associated viral vectors in the treatment of
hemophilia A. Because of the size constraints of recombinant adeno-associated viral …
hemophilia A. Because of the size constraints of recombinant adeno-associated viral …
High-level tissue-specific expression of functional human factor VIII in mice
S Connelly, JM Gardner, A McClelland… - Human Gene …, 1996 - liebertpub.com
Hemophilia A results from subnormal levels of blood coagulation factor VIII (FVIII) and is an
attractive target for gene therapy. However, progress has been impeded by features of FVIII …
attractive target for gene therapy. However, progress has been impeded by features of FVIII …