Results 201 to 210 of about 301,643 (264)

The genetic analysis of eight families with hemophilia B in Mongolia: Identification of two novel mutation. [PDF]

Mol Genet Genomic Med
Munkhuu P, Bazarragchaa M, Ichinkhorloo P, Yoo KY, Ayush EA, Batjargal O, Namjil E, Jav S, Purevdorj E, Lkhagvasuren S.   +9 more
europepmc   +1 more source

Clinical data on treatment regimen and use of medication among patients with hemophilia B in Korea. [PDF]

Blood Res
Park YS, Park JK, Park JA, Baek HJ, Lee JH, You CW, Lyu CJ, Choi EJ.   +7 more
europepmc   +1 more source

118. Incorporation of a Human Replication Signal and a Superactive Factor IX Variant Stablizes and Enhances Long-Term Functional Activities Following Nonviral Gene Transfer in Hemophilia B Mice [PDF]

, 2008

openalex   +1 more source

A transposable element prevents severe hemophilia B and provides insights into the evolution of new- and old world primates. [PDF]

PLoS One
Kopp J, Rovai A, Ott M, Wedemeyer H, Tiede A, Böhmer HJ, Marques T, Langemeier J, Bohne J, Krooss SA.   +9 more
europepmc   +1 more source

125. Towards a New Gene Therapy Approach for Hemophilia B Using microRNA-Regulated Lentiviral Vectors [PDF]

, 2008

openalex   +1 more source

Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec. [PDF]

Blood Adv
Anguela XM, High KA.
europepmc   +1 more source

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

, 2004
Valder R. Arruda, Joerg Schuettrumpf, Roland W. Herzog, Timothy C. Nichols, Nancy M. Robinson, Yasmin Lotfi, Federico Mingozzi, Weidong Xiao, Linda B. Couto, Katherine A. High   +9 more
openalex   +2 more sources

643. Identification of Transgene-Specific CD4+FOXP3+ T Cells Secreting High Levels of IL-10 Following Intravascular AAV-cF.IX Gene Transfer to Muscle in Hemophilia B Dogs [PDF]

, 2009

openalex   +1 more source

Pharmacokinetic model-based assessment of factor IX prophylaxis treatment regimens in severe hemophilia B. [PDF]

Sci Rep
Vandewalle B, Castaman G, Álvarez-Román MT, Ettingshausen CE, Nemes L, Tomic R, Martins P, Rodrigues JF, Pinachyan K.   +8 more
europepmc   +1 more source

603. Correction of Hemophilia B Phenotype by Novel Method of Regional Intravenous Delivery of AAV Vector to Skeletal Muscle of Hemophilia B Dogs

, 2005
Valder R. Arruda, Hansell H. Stedman, Haiyan Jian, Glenn F. Pierce, Joerg Schuettrumpf, Christian Furlan Freguia, Stefano Baila, Kapil Gopal, Timothy C. Nichols, Katherine A. High   +9 more
openalex   +1 more source