Results 231 to 240 of about 50,045 (313)

Damoctocog Alfa Pegol, a PEGylated B-domain Deleted Recombinant Extended Half-life Factor VIII for the Treatment of Hemophilia A: A Product Review [PDF]

Mark T. Reding, Shadan Lalezari, Gili Kenet, Giovanni Di Minno, Jonathan M. Ducore, Alexander Solms, Anita Shah, Pål André Holme, Lone Hvitfeldt Poulsen, Karina Meijer, Mindy Simpson, Maria Elisa Mancuso   +11 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

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

Lentiviral-Engineered Mesenchymal Stem Cells for Hemophilia B Gene Therapy

, 2013
Megan Dodd
openalex   +1 more source

Gene therapy for hemophilia b using pinctm formulated plasmid delivered to muscle with electroporation

, 2000
L. C. Smith, Jason G. Fewell, F.M. MacLaughlin, Vidya Mehta, M. Gondo, François Nicol, Edward M. Nolan, Marjorie S. Read, Timothy C. Nichols, Jeffrey L. Nordstrom   +9 more
openalex   +1 more source

[Gene therapy marks the beginning of a potential "clinical cure" for hemophilia B patients]. [PDF]

Zhonghua Xue Ye Xue Za Zhi
Zhang L.
europepmc   +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

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

Multimodal mechanisms of pathogenic variants in the signal peptide of FIX leading to hemophilia B. [PDF]

Blood Adv
Gao M, Chen L, Yang J, Dong S, Cao Q, Cui Z, Dong Y, Liu H, Shen Y, Yang H, Hao Z, Zhang L, Li W, Tie JK, Shen G.   +14 more
europepmc   +1 more source