Results 211 to 220 of about 13,000,271 (286)

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

, 2008

openalex   +1 more source

Long‐term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

Journal of Controlled Release, 2019
C. Stephens, Elvin J Lauron, E. Kashentseva, Z. Lu, W. Yokoyama, D. Curiel   +5 more
semanticscholar   +1 more source

Retrospective analysis of hemophilia B in Turkey: identifying main characteristics and treatment options. [PDF]

Res Pract Thromb Haemost
Zülfikar B, Koç B, Şahin F, Şaşmaz Hİ, Kavaklı K, Balkan C, Antmen AB, Akbayram S, Güvenç B, Okan V, Türkkan E, Albayrak C, Albayrak D, Sarper N, Celkan TT, Ayyıldız O, Aksu S, Patıroğlu T, Şalcıoğlu Z, Güneş AM, Torun YA, Çalışkan Ü, Tokgöz H, Ay Y, Özdemir GN, Sönmez M, Ünal E, Öner AF, Güler N, Küpesiz OA, Ören H, Karaman S, Ünüvar A, Dağlı M, Demir AM, Söker M, Alioğlu B, Kaya Z, Ayhan AC, Bıçakçı Z, Aral YZ, Ar MC.   +41 more
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

Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B [PDF]

, 2009
Nicola Brunetti‐Pierri, Nathan C. Grove, Yu Zuo, Rachel Edwards, Donna Palmer, Vincenzo Cerullo, Jun Teruya, Philip Ng   +7 more
openalex   +1 more source

Liver-directed lentiviral gene therapy in a dog model of hemophilia B

Science Translational Medicine, 2015
Alessio Cantore, M. Ranzani, C. Bartholomae, M. Volpin, P. Valle, F. Sanvito, L. Sergi, P. Gallina, F. Benedicenti, Dwight Bellinger, R. Raymer, E. Merricks, F. Bellintani, S. Martin, C. Doglioni, A. D’Angelo, T. VandenDriessche, M. Chuah, M. Schmidt, T. Nichols, E. Montini, L. Naldini   +21 more
semanticscholar   +1 more source

AAV vectors for long-term gene therapy of hemophilia B: Are we there yet? [PDF]

Mol Ther
Srivastava A.
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

A Case of Epidural Hematoma after Lumbar Spine Surgery in a Hemophilia B Carrier. [PDF]

JMA J
Sakamoto Y, Ozaki T, Tahata S, Fujimoto T, Naruo S.   +4 more
europepmc   +1 more source

Nonacog beta pegol in previously treated children with hemophilia B: results from an international open‐label phase 3 trial

Journal of Thrombosis and Haemostasis, 2016
M. Carcao, M. Zak, F. Abdul Karim, H. Hanabusa, S. Kearney, M. Lu, P. Persson, S. Rangarajan, E. Santagostino   +8 more
semanticscholar   +1 more source