Results 31 to 40 of about 14,739,736 (367)

CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

EMBO Molecular Medicine, 2016
The X‐linked genetic bleeding disorder caused by deficiency of coagulator factor IX, hemophilia B, is a disease ideally suited for gene therapy with genome editing technology. Here, we identify a family with hemophilia B carrying a novel mutation, Y371D,
Yuting Guan, Yanlin Ma, Qi Li, Zhenliang Sun, Lie Ma, Lijuan Wu, Liren Wang, L. Zeng, Yanjiao Shao, Yuting Chen, Ning Ma, Wenqing Lu, Kewen Hu, Honghui Han, Yanhong Yu, Yuanhua Huang, Mingyao Liu, Dali Li   +17 more
semanticscholar   +1 more source

Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia B

Journal of Blood Medicine, 2023
Katsuyuki Fukutake,1,2 Kanae Togo,3 Linghua Xu,3 Leona E Markson,4 José Maria Jimenez Alvir,5 Ian Winburn,6 Toshiyuki Karumori3 1Laboratory Medicine, Tokyo Medical University, Shinjuku, Tokyo, Japan; 2Department of Blood Coagulation Diseases, Ogikubo ...
Fukutake K, Togo K, Xu L, Markson LE, Alvir JMJ, Winburn I, Karumori T   +6 more
doaj  

Triton Electric Form Factor at Low-Energies [PDF]

, 2009
Making use of the Effective Field Theory(EFT) expansion recently developed by the authors, we compute the charge form factor of triton up to next-to-next-to-leading order (N$^2$LO).
Hossein Sadeghi, Jafar Nezamdost
core   +1 more source

Factor IX p.A37V mutation causes severe bleeding in a patient with phenprocoumon therapy

European Journal of Medical Research, 2021
Background Bleeding is the most common complication of oral anticoagulants, due to inadequate dosing. Case presentation This report describes the clinical course of a patient who developed severe bleeding under therapy with phenprocoumon, despite an INR ...
Nils Mülling, Vivian Rosery, H. Christian Reinhardt, Maher Hanoun   +3 more
doaj   +1 more source

Spin susceptibility of neutron matter at zero temperature [PDF]

, 2001
The Auxiliary Field Diffusion Monte Carlo method is applied to compute the spin susceptibility and the compressibility of neutron matter at zero temperature.
A. Akmal, A. Burrows, A. D. Jackson, A. Sarsa, A. Smerzi, B. L. Friman, G. G. Raffelt, J. W. Clark, K. E. Schmidt, K. E. Schmidt, K. E. Schmidt, N. Iwamoto, R. B. Wiringa, R. F. Sawyer, R. F. Sawyer, R. V. Reid, S. C. Pieper, S. Fantoni, S. Fantoni, S. Fantoni, S. Fantoni, S. O. Bäckmann, S. Reddy   +22 more
core   +4 more sources

Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity.

JCI Insight, 2019
Adeno-associated-viral (AAV) vector liver-directed gene therapy (GT) for hemophilia B (HB) is limited by a vector-dose-dependent hepatotoxicity. Recently, this obstacle has been partially circumvented by the use of a hyperactive factor IX (FIX) variant ...
Benjamin J. Samelson-Jones, Jonathan D. Finn, Lindsey A. George, R. Camire, V. Arruda   +4 more
semanticscholar   +1 more source

Gene Therapy and Hemophilia: Where Do We Go from Here?

Journal of Blood Medicine, 2022
Nancy S Bolous,1 Nidhi Bhatt,2 Nickhill Bhakta,1 Ellis J Neufeld,2 Andrew M Davidoff,3 Ulrike M Reiss2 1Department of Global Pediatric Medicine – St.
Bolous NS, Bhatt N, Bhakta N, Neufeld EJ, Davidoff AM, Reiss UM   +5 more
doaj  

An expression signature of the angiogenic response in gastrointestinal neuroendocrine tumours: correlation with tumour phenotype and survival outcomes. [PDF]

, 2013
BACKGROUND: Gastroenteropancreatic neuroendocrine tumours (GEP-NETs) are heterogeneous with respect to biological behaviour and prognosis. As angiogenesis is a renowned pathogenic hallmark as well as a therapeutic target, we aimed to investigate the ...
A Couvelard, A Giatromanolaki, A Rinke, AM Marion-Audibert, AT Dimou, B Terris, D Hanahan, D J Pinato, D O’Toole, DC Metz, DJ Pinato, DJ Pinato, E Raymond, EP Hui, F Panzuto, G Kloppel, HS Kim, I Albrecht, IM Modlin, J LeCouter, J Strosberg, J Zhang, JC Yao, JT Chi, JY Scoazec, K Hirabayashi, K Meeran, K Villaume, M Dal Monte, M Perigny, M Ruscica, MA Maynard, N Martin, N Ngo, P Kuiper, R Dina, R Katoh, R Mentlein, R Ramachandran, R Sharma, S Ezziddin, S T K Toussi, T M Tan, VD Corleto, Y Arvidsson, Y Seo, Y Takahashi, YC Patel   +47 more
core   +2 more sources

Electromagnetic Structure of the Trinucleons [PDF]

, 1998
The electromagnetic form factors of the trinucleons 3H and 3He are calculated with wave functions obtained with the Argonne AV18 two-nucleon and Urbana IX three-nucleon interactions.
Marcucci, L. E., Riska, D. O., Schiavilla, R.   +2 more
core   +3 more sources

Seroprevalence of Hepatitis B and Hepatitis C in People with Haemophilia at a Tertiary Care Centre, Assam, India: A Cross-sectional Study [PDF]

Journal of Clinical and Diagnostic Research
Introduction: Prior to the advent of recombinant factor products, the treatment of haemophilia was mainly based on the administration of direct blood products like whole blood, Fresh Frozen Plasma (FFP), and cryoprecipitates. This led to a high incidence
Anupam Dutta, Luish Borboruah, Papori Gogoi   +2 more
doaj   +1 more source