Results 51 to 60 of about 50,045 (313)
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 +6 more
wiley +1 more source
Queen Victoria and The Royal Disease [PDF]
, 2018 Queen Victoria, Queen of England and Ireland and Empress of India, is the second longest ruling monarch of England, she sat on the throne for sixty –three years and was so crucial to the formation of England that there is a whole era names after her, The
Mullen, Madeline
core +1 more source
Disclosure of Maternal HIV Status to Children: To Tell or Not To Tell . . . That Is the Question [PDF]
, 2007 HIV-infected mothers face the challenging decision of whether to disclose their serostatus to their children. From the perspective of both mother and child, we explored the process of disclosure, providing descriptive information and examining the ...
A. Shaffer +46 more
core +3 more sources
Annals of Neurology, EarlyView.Pathogenic DMD variants usually follow the reading‐frame rule: out‐of‐frame changes cause Duchenne muscular dystrophy, whereas in‐frame ones produce Becker muscular dystrophy (BMD). We report a 23‐year‐old man with BMD‐like weakness, calf hypertrophy, elevated creatine kinase, and dilated cardiomyopathy.
Hiroya Naruse +16 more
wiley +1 more source
EMBO Molecular Medicine, 2016 Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B.
Tuan Huy Nguyen, Ignacio Anegon
doaj +1 more source
Journal of Family Medicine and Primary Care, 2019 Introduction: Deficiency of factor VIII (Hemophilia A), factor IX (Hemophilia B) and Von Willebrand's factor are the most frequent coagulation defects.
Sandip Kumar +5 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. +16 more
core
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
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
Strategies for Hemophilia Treatment, a literature review of current evidence
مجله دانشگاه علوم پزشکی بیرجند, 2023 Hemophilia is an inherited bleeding disorder caused by malfunctioning or lacking blood coagulation factor VIII (hemophilia A) or IX (hemophilia B). Currently, the main treatments for these X-linked diseases are replacement therapy using periodic and ...
Fahimeh Ghasemi +3 more
doaj