Results 191 to 200 of about 13,000,271 (286)

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy [PDF]

, 2005
Lili Wang, Roberto Calcedo, Timothy C. Nichols, Dwight A. Bellinger, Aaron Dillow, Inder M. Verma, James M. Wilson   +6 more
openalex   +1 more source

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

Molecular Therapy: Methods & Clinical Development, 2019
A. Majowicz, B. Nijmeijer, M. Lampen, Lisa Spronck, M. de Haan, H. Petry, S. V. van Deventer, C. Meyer, M. Tangelder, Valérie Ferreira   +9 more
semanticscholar   +1 more source

Trenonacog alfa safety, efficacy, and pharmacokinetics in previously treated pediatric hemophilia B. [PDF]

Res Pract Thromb Haemost
Vilchevska K, Balkan C, Turea V, Gurtchumelia D, Loureiro Prezotti AN, Oliveira de Oliveira LC, Leon J, Fosdal M, Mahlangu J.   +8 more
europepmc   +1 more source

111. AAV7, 8, and 9 Are More Efficient and Less Immunogenic Vectors for Muscle-Directed Gene Therapy for Hemophilia B [PDF]

, 2005
Lin‐Fa Wang, Jean-Pierre Louboutin, Yan Li, James M. Wilson   +3 more
openalex   +1 more source

Characteristics of FXa-storing platelets in hemophilia B mice and the influence of alcohol on the platelets. [PDF]

Sci Rep, 2023
Han W, Huang R, Li B, Liu L, Xu W, Zhang G.   +5 more
europepmc   +1 more source

32. Tolerance after Neonatal Gene Transfer of a Human Factor IX-Expressing Retroviral Vector May Involve Clonal Deletion in C3H Mice and Is Effective in Hemophilia B Dogs [PDF]

, 2005
Lingfei Xu, Mark E. Haskins, Timothy C. Nichols, Manxue Mei, Patty O'Donnell, Dwight A. Bellinger, Stephanie McCorquodale, Katherine P. Ponder   +7 more
openalex   +1 more source

Preclinical Evaluation of the Systemic Safety, Efficacy, and Biodistribution of a Recombinant AAV8 Vector Expressing FIX-TripleL in Hemophilia B Mice: Implications for Human Gene Therapy. [PDF]

Int J Mol Sci
Chou SC, Huang CP, Su YH, Yu CH, Yang YL, Wang SC, Lin YH, Chen YT, Li JY, Chang YT, Chen SY, Lin SW.   +11 more
europepmc   +1 more source

Lipid Nanoparticle-Mediated Liver-Specific Gene Therapy for Hemophilia B. [PDF]

Pharmaceutics
Lohchania B, Arjunan P, Mahalingam G, Dandapani A, Taneja P, Marepally S.   +5 more
europepmc   +1 more source

Use of nonacog beta pegol during surgery in persons with hemophilia B: a case series. [PDF]

Res Pract Thromb Haemost, 2023
Phua CW, Matino D, Kühnöl CD, Hegemann I, Matsushita T.   +4 more
europepmc   +1 more source

A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice [PDF]

, 2007
Brian D. Brown, Alessio Cantore, Andrea Annoni, Lucia Sergi Sergi, Angelo Lombardo, Patrizia Della Valle, Armando D’Angelo, Luigi Naldini   +7 more
openalex   +1 more source