Results 61 to 70 of about 13,086,034 (342)

Recurrent bleeding after rubber band ligation diagnosed as mild hemophilia B: a case report and literature review

BMC Surgery, 2022
Background Hemophilia is a recessive hemorrhagic disease relevant to X chromosome. In mild hemophilia cases, spontaneous bleeding is rare and the blood clotting function is normal, but severe bleeding may occur after trauma or surgery.
Xiaoying Jiang, Min Xu, Yaqing Ding, Yongqing Cao, Yibin Pan   +4 more
doaj   +1 more source

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Blood, 2018
Background: We have previously shown that a single intravenous administration of a self-complementary adeno-associated virus (scAAV) vector containing a codon-optimised factor IX gene, under control of a synthetic liver specific promoter and ...
A. Nathwani, U. Reiss, E. Tuddenham, P. Chowdary, J. McIntosh, A. Riddell, J. Pie, Johnny Mahlangu, M. Recht, Yu-min P Shen, K. Halka, M. Meagher, A. Nienhuis, A. Davidoff, S. Mangles, C. Morton, Junfang Zhou, V. Radulescu   +17 more
semanticscholar   +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

Serum Vitamin D in Children with Hemophilia A and Its Association with Joint Health and Quality of Life

Hematology Reports
Background/Objectives: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of ...
Aida M. S. Salem, Takwa Mohamed AbdEltwwab, Hanan Hosni Moawad, Marwa O. Elgendy, Reham S. Al-Fakharany, Ahmed Khames, Mohamed Hussein Meabed   +6 more
doaj   +1 more source

In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A. [PDF]

, 2018
Hemophilia A (HA) is an X-linked recessive disorder caused by mutations in the factor VIII ( FVIII) gene leading to deficient blood coagulation. The current standard of care is frequent infusions of plasma-derived FVIII or recombinant B-domain-deleted ...
Farmer, Diana, Gao, Kewa, Kumar, Priyadarsini, Lankford, Lee, Pivetti, Christopher, Wang, Aijun, Wang, Chuwang   +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, 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

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

PRODUCTION OF HIGH LEVELS OF TRANSGENC FACTOR IX WITHOUT GENE RESCUE, AND ITS THERAPEUTIC USES [PDF]

, 2011
A non-human transgenic mammalian animal, as described above, contains an exogenous double stranded DNA sequence stably integrated into the genome of the animal, which comprises cis-acting regulatory units operably linked to a DNA sequence encoding human ...
Cooper, Julian D., O\u27Sickey, Tanya K., Velander, William Hugold   +2 more
core   +1 more source

An assessment of the relative influence of pain coping, negative thoughts about pain, and pain acceptance on health-related quality of life among people with hemophilia [PDF]

, 2009
Many people with hemophilia are affected by chronic arthritic joint pain as well as acute bleeding pain. In this cross-sectional study, 209 men with hemophilia A or B completed the Hemophilia Pain Coping Questionnaire (HPCQ), the Chronic Pain Acceptance ...
Barakat, Baron, Barry, Choiniere, Elander, Elander, Esteve, Fischer, Fischer, Georgina Robinson, Gil, Gil, Hays, Hays, James Elander, Jensen, Jensen, John Morris, Kathryn Mitchell, Kratz, McCracken, McCracken, McCracken, McCracken, McCracken, McCracken, McCracken, McHorney, McHorney, Miners, Miners, Preacher, Roberts, Romano, Rosenstiel, Royal, Ruta, Santavirta, Solovieva, Solovieva, Stansfield, Szende, Trippoli, Turner, Turner, Vowles, Vowles, Vowles   +47 more
core   +1 more source