Results 41 to 50 of about 68,267 (269)
Diagnosis of von Willebrand disease in Argentina: a single institution experience [PDF]
, 2017 von Willebrand disease (VWD) is the most common autosomal bleeding disorder, mostlyinherited as dominant trait. VWD is due to deficiency/abnormality of von Willebrand factor (VWF).
Blanco, Alicia Noemi +5 more
core +1 more source
Biotechnology and Bioengineering, EarlyView.ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai +9 more
wiley +1 more source
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 +8 more
doaj +1 more source
Hypoxic Preconditioning Enhances Survival and Proangiogenic Capacity of Human First Trimester Chorionic Villus-Derived Mesenchymal Stem Cells for Fetal Tissue Engineering. [PDF]
, 2019 Prenatal stem cell-based regenerative therapies have progressed substantially and have been demonstrated as effective treatment options for fetal diseases that were previously deemed untreatable. Due to immunoregulatory properties, self-renewal capacity,
Farmer, Diana L +7 more
core +1 more source
A Mechanistic Model of rAAV Production in Synthetic Cell Lines
Biotechnology and Bioengineering, EarlyView.ABSTRACT The recombinant adeno‐associated virus (rAAV) is a widely used vector for gene therapy. Its manufacturing faces significant challenges in producing the large quantities of vectors needed for clinical applications and reducing empty particles.
Prahalad Srinivasan +5 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 +4 more
doaj +1 more source
Clinical and Applied Thrombosis/Hemostasis, 2023 In 2018, Refacto AF R , a B-domain-deleted third-generation FVIII concentrate, became our preferential product. After the introduction, the development of inhibitors was prospectively monitored; retrospectively, we sought for risk factors in the patients
Louise H Hooimeijer MD +6 more
doaj +1 more source
Perinatal Gene Transfer to the Liver [PDF]
, 2011 The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM +6 more
core +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 +8 more
wiley +1 more source
Journal of Orthopaedic Surgery and Research, 2022 Background Although arthroplasty provides satisfactory pain relief, functional improvement, and reduced flexion contracture in patients with hemophilia arthropathy, bleeding remains the primary problem associated with hemophilic arthropathy joint ...
Shanyou Yuan +5 more
doaj +1 more source