Results 41 to 50 of about 83,381 (315)

Construction of pathogenic Sec16a mutation mouse model using CRISPR/Cas9

Animal Models and Experimental Medicine, EarlyView.
Yaqiang Hu et al. engineered a pathogenic Sec16a mutant mouse model using CRISPR/Cas9 technology. They observed that the Sec16a mutant mice displayed diminished learning and memory capabilities, along with a limb‐clasping phenotype upon tail suspension.
Yaqiang Hu, Zhiyang Zeng, Xinyu Ming, Shuming Yin, Yuting Guan, Liangcai Gao, Dali Li   +6 more
wiley   +1 more source

Correlation between APTT mixing test results and inhibitor titers in patients with hemophilia A inhibitor

Zhongguo shuxue zazhi
[Objective] To study the correlation between activated partial thromboplastin time (APTT) mixing test results and the inhibitor titers in hemophilia A inhibitor-positive patients.
WANG Tiantian, CHENG Yan, WANG Hehe, QIAO Cuicui, WANG Jie, ZHONG Guangshang, WANG Xiyuan, ZHANG Xueqin, FANG Yunhai   +8 more
doaj   +1 more source

Managing Relevant Clinical Conditions of Hemophilia A/B Patients

Hematology Reports, 2023
The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini, Jacopo Agnelli Giacchiello, Erminia Baldacci, Christian Carulli, Giancarlo Castaman, Anna Chiara Giuffrida, Giuseppe Malcangi, Angiola Rocino, Sergio Siragusa, Ezio Zanon   +9 more
doaj   +1 more source

M/G‐Ratio‐Tuned Calcium Alginate Hydrogel Microcarrier for Controlled Delivery of Adeno‐Associated Virus for Gene Therapy

Biotechnology and Bioengineering, EarlyView.
ABSTRACT This paper proposes the controlled release of adeno‐associated virus (AAV) by polymer mesh structures of alginate hydrogel microbeads (gel‐beads). The polymer mesh structure of alginate hydrogel can be controlled by adjusting the number of cross‐linking points depending on the molecular structure (M/G ratio) of the alginate hydrogel.
Aiki Hioki, Shuhei Takatsuka, Yuta Kurashina, Hiroaki Onoe   +3 more
wiley   +1 more source

Retrospective analysis of clinical data from 171 low-income patients with hemophilia in Shandong Province

Scientific Reports
To enhance attention on low-income patients with hemophilia, it is essential to urge governments and coagulation factor manufacturers to increase their investment in hemophilia care.
Jie Wang, Yan Cheng, Yunhai Fang, Cuicui Qiao, Tiantian Wang, Hehe Wang, Xueqin Zhang, Qiang Li   +7 more
doaj   +1 more source

Optimization of CEST MRI Reporter Protein Design Using Cation‐Pi Networks

Chemistry – A European Journal, EarlyView.
A novel engineering approach can produce reporter proteins for cell and viral therapy tracking with unique magnetic resonance imaging (MRI) signatures, detectable with chemical exchange saturation transfer (CEST). We discover how cation‐π interactions between amino acid groups can help us fine‐tune magnetic resonance properties for noninvasive ...
David E. Korenchan, Ethan J. French, Emerenziana Runco, Chetan B. Dhakan, Jinwu Yan, Hiroshi Nakashima, Michael T. McMahon, Assaf A. Gilad, Christian T. Farrar   +8 more
wiley   +1 more source

The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B

Blood Advances, 2018
: Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
J. Michael Soucie, Paul E. Monahan, Roshni Kulkarni, Barbara A. Konkle, Marshall A. Mazepa   +4 more
doaj   +1 more source

A Comparison between Recombinant Activated Factor VII (Aryoseven) and Novoseven in Patients with Congenital Factor VII Deficiency [PDF]

, 2015
In order to establish the efficacy and biosimilar nature of AryoSeven to NovoSeven in the treatment of congenital factor VII (FVII) deficiency, patients received either agent at 30 1/4g/kg, intravenously per week for 4 weeks, in a randomized fashion. The
Abolghasemi, H., Ahmadinejad, M., Baghaeipour, M.R., Dehdezi, B.K., Eshghi, P., Faranoush, M., Fayazfar, R., Heshmat, R., Hoorfar, H., Kamyar, K., Karimi, M., Khoeiny, B., Managhchi, M., Mehrvar, A., Mirbehbahani, N.B., Naderi, M., Toogeh, G.   +16 more
core  

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Model‐Informed Drug Development Applications and Opportunities in mRNA‐LNP Therapeutics

Clinical Pharmacology &Therapeutics, EarlyView.
The utilization of lipid nanoparticles (LNP) for encapsulating mRNA has revolutionized the field of therapeutics, enabling the rapid development of COVID‐19 vaccines and cancer vaccines. However, the clinical development of mRNA‐LNP therapeutics faces numerous challenges due to their complex mechanisms of action and limited clinical experience.
Jiawei Zhou, Rohit Rao, Monica E. Shapiro, Nessy Tania, Cody Herron, Cynthia J. Musante, Jim H. Hughes   +6 more
wiley   +1 more source