Search Results - "Couto, Linda B"

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy by Jiang, Haiyan, Couto, Linda B., Patarroyo-White, Susannah, Liu, Tongyao, Nagy, Dea, Vargas, Joseph A., Zhou, Shangzhen, Scallan, Ciaran D., Sommer, Jurg, Vijay, Sharmila, Mingozzi, Federico, High, Katherine A., Pierce, Glenn F.

“…In a clinical study of recombinant adeno-associated virus-2 expressing human factor IX (AAV2-FIX), we detected 2 impediments to long-term gene transfer. First,…”
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Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice by Scallan, Ciaran D., Jiang, Haiyan, Liu, Tongyao, Patarroyo-White, Susannah, Sommer, Jurg M., Zhou, Shangzhen, Couto, Linda B., Pierce, Glenn F.

“…Long-term cures of hemophilia B have been achieved using AAV2 delivering the factor IX gene to the liver of adeno-associated virus (AAV)–naive hemophilic…”
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Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B by Arruda, Valder R., Stedman, Hansell H., Haurigot, Virginia, Buchlis, George, Baila, Stefano, Favaro, Patricia, Chen, Yifeng, Franck, Helen G., Zhou, Shangzhen, Wright, J. Fraser, Couto, Linda B., Jiang, Haiyan, Pierce, Glenn F., Bellinger, Dwight A., Mingozzi, Federico, Nichols, Timothy C., High, Katherine A.

“…Muscle represents an important tissue target for adeno-associated viral (AAV) vector-mediated gene transfer of the factor IX (FIX) gene in hemophilia B (HB)…”
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Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs by Jiang, Haiyan, Lillicrap, David, Patarroyo-White, Susannah, Liu, Tongyao, Qian, Xiaobing, Scallan, Ciaran D., Powell, Sandra, Keller, Tracey, McMurray, Morag, Labelle, Andrea, Nagy, Dea, Vargas, Joseph A., Zhou, Shangzhen, Couto, Linda B., Pierce, Glenn F.

“…Hemophilia A, a deficiency of functional coagulation factor VIII (FVIII), is treated via protein replacement therapy. Restoring 1% to 5% of normal blood FVIII…”
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Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector by High, Katherine A, Herzog, Roland W, Yang, Edmund Y, Couto, Linda B, Hagstrom, J. Nathan, Elwell, Dan, Fields, Paul A, Burton, Melissa, Bellinger, Dwight A, Read, Marjorie S, Brinkhous, Kenneth M, Podsakoff, Gregory M, Nichols, Timothy C, Kurtzman, Gary J

“…Hemophilia B is a severe X-linked bleeding diathesis caused by the absence of functional blood coagulation factor IX, and is an excellent candidate for…”
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant by George, Lindsey A, Sullivan, Spencer K, Giermasz, Adam, Rasko, John E.J, Samelson-Jones, Benjamin J, Ducore, Jonathan, Cuker, Adam, Sullivan, Lisa M, Majumdar, Suvankar, Teitel, Jerome, McGuinn, Catherine E, Ragni, Margaret V, Luk, Alvin Y, Hui, Daniel, Wright, J. Fraser, Chen, Yifeng, Liu, Yun, Wachtel, Katie, Winters, Angela, Tiefenbacher, Stefan, Arruda, Valder R, van der Loo, Johannes C.M, Zelenaia, Olga, Takefman, Daniel, Carr, Marcus E, Couto, Linda B, Anguela, Xavier M, High, Katherine A

“…An adeno-associated viral vector was used to introduce a FIX gene with enhanced biologic activity in 10 participants with hemophilia B. The annualized bleeding…”
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Viral vector-mediated RNA interference by Couto, Linda B, High, Katherine A

“…RNA interference (RNAi) is a powerful gene silencing mechanism that if properly harnessed has the potential to revolutionize medical interventions. Delivery of…”
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Posttranslational modifications of recombinant myotube-synthesized human factor IX by Arruda, Valder R., Hagstrom, James N., Deitch, Jeffrey, Heiman-Patterson, Terry, Camire, Rodney M., Chu, Kirk, Fields, Paul A., Herzog, Roland W., Couto, Linda B., Larson, Peter J., High, Katherine A.

“…Recent data demonstrate that the introduction into skeletal muscle of an adeno-associated viral (AAV) vector expressing blood coagulation factor IX (F.IX) can…”
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Preclinical Evaluation of An Anti-HCV miRNA Cluster for Treatment of HCV Infection by Yang, Xiao, Marcucci, Katherine, Anguela, Xavier, Couto, Linda B.

“…We developed a strategy to treat hepatitis C virus (HCV) infection by replacing five endogenous microRNA (miRNA) sequences of a natural miRNA cluster…”
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Inhibition of hepatitis C virus replication using adeno‐associated virus vector delivery of an exogenous anti–hepatitis C virus microrna cluster by Yang, Xiao, Haurigot, Virginia, Zhou, Shangzhen, Luo, Guangxiang, Couto, Linda B

“…RNA interference (RNAi) is being evaluated as an alternative therapeutic strategy for hepatitis C virus (HCV) infection. The use of viral vectors encoding…”
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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B by Manno, Catherine S., Chew, Amy J., Hutchison, Sylvia, Larson, Peter J., Herzog, Roland W., Arruda, Valder R., Tai, Shing Jen, Ragni, Margaret V., Thompson, Arthur, Ozelo, Margareth, Couto, Linda B., Leonard, Debra G.B., Johnson, Frederick A., McClelland, Alan, Scallan, Ciaran, Skarsgard, Erik, Flake, Alan W., Kay, Mark A., High, Katherine A., Glader, Bertil

“…Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (F.IX). Previously, we established an experimental basis for…”
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Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model by Arruda, Valder R., Stedman, Hansell H., Nichols, Timothy C., Haskins, Mark E., Nicholson, Matthew, Herzog, Roland W., Couto, Linda B., High, Katherine A.

“…In earlier work, we showed that adeno-associated virus–mediated delivery of a Factor IX gene to skeletal muscle by direct intramuscular injection resulted in…”
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Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector by High, Katherine A, Kay, Mark A, Manno, Catherine S, Ragni, Margaret V, Larson, Peter J, Couto, Linda B, McClelland, Alan, Glader, Bertil, Chew, Amy J, J Tai, Shing, Herzog, Roland W, Arruda, Valder, Johnson, Fred, Scallan, Ciaran, Skarsgard, Erik, Flake, Alan W

“…Pre-clinical studies in mice and haemophilic dogs have shown that introduction of an adeno-associated viral (AAV) vector encoding blood coagulation factor IX…”
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Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1 by Arruda, Valder R., Schuettrumpf, Joerg, Herzog, Roland W., Nichols, Timothy C., Robinson, Nancy, Lotfi, Yasmin, Mingozzi, Federico, Xiao, Weidong, Couto, Linda B., High, Katherine A.

“…Adeno-associated viral (AAV) vectors (serotype 2) efficiently transduce skeletal muscle, and have been used as gene delivery vehicles for hemophilia B and for…”
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Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver by Nakai, H, Iwaki, Y, Kay, M A, Couto, L B

“…Article Usage Stats Services JVI Citing Articles Google Scholar PubMed Related Content Social Bookmarking CiteULike Delicious Digg Facebook Google+ Mendeley…”
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Inadvertent Germline Transmission of AAV2 Vector: Findings in a Rabbit Model Correlate with Those in a Human Clinical Trial by Schuettrumpf, Joerg, Liu, Jian-Hua, Couto, Linda B, Addya, Kathakaly, Leonard, Debra G B, Zhen, Zhu, Sommer, Jürg, Summer, Jürg, Arruda, Valder R

“…The risk of germline transmission of vector sequences in humans is a major safety concern, because the enrollment of subjects of reproductive age in…”
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Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy by Herzog, Roland W, Fields, Paul A, Arruda, Valder R, Brubaker, Jeff O, Armstrong, Elina, McClintock, Darryl, Bellinger, Dwight A, Couto, Linda B, Nichols, Timothy C, High, Katherine A

“…Intramuscular injection of an adeno-associated virus (AAV) vector has resulted in vector dose-dependent, stable expression of canine factor IX (cF.IX) in…”
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Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector by Scallan, Ciaran D., Lillicrap, David, Jiang, Haiyan, Qian, Xiaobing, Patarroyo-White, Susannah L., Parker, Amy E., Liu, Tongyao, Vargas, Joseph, Nagy, Dea, Powell, Sharon K., Wright, J. Fraser, Turner, Patricia V., Tinlin, Shawn J., Webster, Sandra E., McClelland, Alan, Couto, Linda B.

“…Gene therapy for hemophilia A requires efficient delivery of the factor VIII gene and sustained protein expression at circulating levels of at least 1% to 2%…”
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Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII by Scallan, Ciaran D., Liu, Tongyao, Parker, Amy E., Patarroyo-White, Susannah L., Chen, Haifeng, Jiang, Haiyan, Vargas, Joseph, Nagy, Dea, Powell, Sharon K., Wright, J. Fraser, Sarkar, Rita, Kazazian, Haig H., McClelland, Alan, Couto, Linda B.

“…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…”
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Preclinical gene therapy studies for hemophilia using adeno-associated virus (AAV) vectors by Couto, Linda B

“…Gene therapy offers a potential cure for hemophilia and several gene transfer vectors have been evaluated for their ability to treat this disease. This article…”
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