A5048635228- Blood Coagulation and Thrombosis Mechanisms
- Cancer-related gene regulation
- Hemophilia Treatment and Research
- Genomics and Rare Diseases
- Acute Myocardial Infarction Research
- Blood disorders and treatments
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- RNA regulation and disease
- Lipoproteins and Cardiovascular Health
- Platelet Disorders and Treatments
- RNA and protein synthesis mechanisms
- RNA modifications and cancer
- Virus-based gene therapy research
- Signaling Pathways in Disease
- Neonatal and Maternal Infections
- Biochemical and Molecular Research
- Protease and Inhibitor Mechanisms
- Mechanical Circulatory Support Devices
- CRISPR and Genetic Engineering
- Heparin-Induced Thrombocytopenia and Thrombosis
- Chemical Synthesis and Analysis
- Venomous Animal Envenomation and Studies
- Computational Drug Discovery Methods
- RNA Research and Splicing
Kanazawa University
2019-2025
Jichi Medical University
2023-2024
Tochigi Medical Center
2023
Industrial Research Institute of Ishikawa
2023
SpCas9 and AsCas12a are widely utilized as genome-editing tools in human cells. However, their relatively large size poses a limitation for delivery by cargo-size-limited adeno-associated virus (AAV) vectors. The type V-F Cas12f from Acidibacillus sulfuroxidans is exceptionally compact (422 amino acids) has been harnessed tool. Here, we developed an approach, combining deep mutational scanning structure-informed design, to successfully generate two AsCas12f activity-enhanced (enAsCas12f)...
PC (protein C) is a plasma anticoagulant encoded by
Abstract The repair of pathological gene variants is an ultimate aim for treating genetic diseases; however, it not practical to develop different therapeutic reagents each the many that can occur in a gene. Here, we investigated whether base editing induce gain-of-function variant blood coagulation factor IX (FIX) increase FIX activity as treatment strategy hemophilia B. We engineered G:C A:T substitution at c.1151 F9 by cytosine generate R338Q, known Shanghai variant, which markedly...
ABSTRACT Protein C (PC) is a plasma anticoagulant encoded by PROC ; mutation in both alleles results neonatal purpura fulminans—a fatal systemic thrombotic disorder. In the present study, we aimed to develop genome editing treatment cure congenital PC deficiency. First, generated an engineered activated insert self-cleaving peptide sequence between light and heavy chains. The could be released its form significantly prolonged coagulation time independent of cofactor activity protein S vitro...
Introduction: Hereditary antithrombin (AT) deficiency type I causes venous thrombosis due to decreased levels of AT antigen in the blood. We identified one novel and known abnormal variant two Japanese unrelated families with thrombosis. In this study, we analyzed mechanism by which these variants cause deficiency.Materials methods: Wild-type expression vectors were constructed transiently expressed human embryonic kidney 293 cells, N-glycosylation cell lysates culture medium evaluated...