[HTML][HTML] Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B
O Cao, BE Hoffman, B Moghimi, S Nayak, M Cooper… - Molecular Therapy, 2009 - cell.com
Immune responses to factor IX (F. IX), a major concern in gene therapy for hemophilia, were
analyzed for adeno-associated viral (AAV-2) gene transfer to skeletal muscle and liver as a …
analyzed for adeno-associated viral (AAV-2) gene transfer to skeletal muscle and liver as a …
[HTML][HTML] Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9
PA Fields, VR Arruda, E Armstrong, K Chu, F Mingozzi… - Molecular Therapy, 2001 - cell.com
The safety of several gene therapy approaches for treatment of the severe, X-linked
bleeding disorder hemophilia is currently being evaluated in early phase clinical trials. One …
bleeding disorder hemophilia is currently being evaluated in early phase clinical trials. One …
Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy
RW Herzog, PA Fields, VR Arruda, JO Brubaker… - Human gene …, 2002 - liebertpub.com
Intramuscular injection of an adeno-associated virus (AAV) vector has resulted in vector
dose-dependent, stable expression of canine factor IX (cF. IX) in hemophilia B dogs with an …
dose-dependent, stable expression of canine factor IX (cF. IX) in hemophilia B dogs with an …
[HTML][HTML] Persistent expression of hF. IX After tolerance induction by in utero or neonatal administration of AAV-1-F. IX in hemophilia B mice
DE Sabatino, TC MacKenzie, W Peranteau… - Molecular Therapy, 2007 - cell.com
The major complication associated with protein replacement therapy currently used in the
treatment of hemophilia B (HB) is the development of antibodies to the infused human Factor …
treatment of hemophilia B (HB) is the development of antibodies to the infused human Factor …
[HTML][HTML] Prevention and reversal of antibody responses against factor IX in gene therapy for hemophilia B
S Nayak, D Sarkar, GQ Perrin, B Moghimi… - Frontiers in …, 2011 - frontiersin.org
Intramuscular (IM) administration of an adeno-associated viral (AAV) vector represents a
simple and safe method of gene transfer for treatment of the X-linked bleeding disorder …
simple and safe method of gene transfer for treatment of the X-linked bleeding disorder …
Factor IX variants improve gene therapy efficacy for hemophilia B
J Schuettrumpf, RW Herzog, A Schlachterman… - Blood, 2005 - ashpublications.org
Intramuscular injection of adeno-associated viral (AAV) vector to skeletal muscle of humans
with hemophilia B is safe, but higher doses are required to achieve therapeutic factor IX (F …
with hemophilia B is safe, but higher doses are required to achieve therapeutic factor IX (F …
Muscle as a target for supplementary factor IX gene transfer
BE Hoffman, E Dobrzynski, L Wang, L Hirao… - Human gene …, 2007 - liebertpub.com
Immune responses to the factor IX (F. IX) transgene product are a concern in gene therapy
for the X-linked bleeding disorder hemophilia B. The risk for such responses is determined …
for the X-linked bleeding disorder hemophilia B. The risk for such responses is determined …
[HTML][HTML] Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX
PA Fields, DW Kowalczyk, VR Arruda, E Armstrong… - Molecular Therapy, 2000 - cell.com
Defining immune responses against the secreted transgene product in a gene therapy
setting is critical for treatment of genetic diseases such as hemophilia B (coagulation factor …
setting is critical for treatment of genetic diseases such as hemophilia B (coagulation factor …
[HTML][HTML] Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation
RW Herzog, JD Mount, VR Arruda, KA High… - Molecular Therapy, 2001 - cell.com
The X-linked bleeding disorder hemophilia B is caused by absence of functional blood
coagulation factor IX (F9) and can be treated by adeno-associated viral (AAV) mediated …
coagulation factor IX (F9) and can be treated by adeno-associated viral (AAV) mediated …
Major role of local immune responses in antibody formation to factor IX in AAV gene transfer
L Wang, O Cao, B Swalm, E Dobrzynski, F Mingozzi… - Gene Therapy, 2005 - nature.com
The risk of an immune response to the coagulation factor IX (F. IX) transgene product is a
concern in gene therapy for the X-linked bleeding disorder hemophilia B. In order to …
concern in gene therapy for the X-linked bleeding disorder hemophilia B. In order to …
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