Pesquisa | BVS Doenças Infecciosas e Parasitárias

1.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

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.

N Engl J Med ; 377(23): 2215-2227, 2017 12 07.

Artigo em Inglês | MEDLINE | ID: mdl-29211678

2.

Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infection.

Yang, Xiao; Marcucci, Katherine; Anguela, Xavier; Couto, Linda B.

Mol Ther ; 21(3): 588-601, 2013 Mar.

Artigo em Inglês | MEDLINE | ID: mdl-23295950

3.

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

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.

Blood ; 115(23): 4678-88, 2010 Jun 10.

Artigo em Inglês | MEDLINE | ID: mdl-20335222

4.

Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microRNA cluster.

Yang, Xiao; Haurigot, Virginia; Zhou, Shangzhen; Luo, Guangxiang; Couto, Linda B.

Hepatology ; 52(6): 1877-87, 2010 Dec.

Artigo em Inglês | MEDLINE | ID: mdl-20931557

5.

Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver.

Ohashi, Kazuo; Nakai, Hiroyuki; Couto, Linda B; Kay, Mark A.

Hum Gene Ther ; 16(3): 299-306, 2005 Mar.

Artigo em Inglês | MEDLINE | ID: mdl-15812225

6.

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy.

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.

Hum Gene Ther ; 13(11): 1281-91, 2002 Jul 20.

Artigo em Inglês | MEDLINE | ID: mdl-12162811

7.

AAV-mediated gene therapy for hemophilia.

Couto, Linda B; Pierce, Glenn F.

Curr Opin Mol Ther ; 5(5): 517-23, 2003 Oct.

Artigo em Inglês | MEDLINE | ID: mdl-14601521

8.

Viral vector-mediated RNA interference.

Couto, Linda B; High, Katherine A.

Curr Opin Pharmacol ; 10(5): 534-42, 2010 Oct.

Artigo em Inglês | MEDLINE | ID: mdl-20620113

9.

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice.

Scallan, Ciaran D; Jiang, Haiyan; Liu, Tongyao; Patarroyo-White, Susannah; Sommer, Jurg M; Zhou, Shangzhen; Couto, Linda B; Pierce, Glenn F.

Blood ; 107(5): 1810-7, 2006 Mar 01.

Artigo em Inglês | MEDLINE | ID: mdl-16249376

10.

Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial.

Schuettrumpf, Joerg; Liu, Jian-Hua; Couto, Linda B; Addya, Kathakaly; Leonard, Debra G B; Zhen, Zhu; Sommer, Jürg; Summer, Jürg; Arruda, Valder R.

Mol Ther ; 13(6): 1064-73, 2006 Jun.

Artigo em Inglês | MEDLINE | ID: mdl-16631412

11.

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs.

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.

Blood ; 108(1): 107-15, 2006 Jul 01.

Artigo em Inglês | MEDLINE | ID: mdl-16522813

12.

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.

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.

Blood ; 108(10): 3321-8, 2006 Nov 15.

Artigo em Inglês | MEDLINE | ID: mdl-16868252

13.

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.

Arruda, Valder R; Stedman, Hansell H; Nichols, Timothy C; Haskins, Mark E; Nicholson, Matthew; Herzog, Roland W; Couto, Linda B; High, Katherine A.

Blood ; 105(9): 3458-64, 2005 May 01.

Artigo em Inglês | MEDLINE | ID: mdl-15479726

14.

Preclinical gene therapy studies for hemophilia using adeno-associated virus (AAV) vectors.

Couto, Linda B.

Semin Thromb Hemost ; 30(2): 161-71, 2004 Apr.

Artigo em Inglês | MEDLINE | ID: mdl-15118928

15.

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.

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.

Blood ; 103(1): 85-92, 2004 Jan 01.

Artigo em Inglês | MEDLINE | ID: mdl-12969984

16.

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII.

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.

Blood ; 102(12): 3919-26, 2003 Dec 01.

Artigo em Inglês | MEDLINE | ID: mdl-12893764

17.

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector.

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.

Blood ; 102(6): 2031-7, 2003 Sep 15.

Artigo em Inglês | MEDLINE | ID: mdl-12738670

18.

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

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.

Blood ; 101(8): 2963-72, 2003 Apr 15.

Artigo em Inglês | MEDLINE | ID: mdl-12515715

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