Results 31 to 40 of about 301,643 (264)
A synthetic platform for developing recombinant adeno‐associated virus type 8 producer cell lines
Biotechnology Progress, EarlyView.Abstract Recombinant adeno‐associated virus (rAAV) is one of the most widely used viral vectors for gene therapy. It is used in very high doses for the treatment of many diseases, making large‐scale production for clinical applications challenging. We have established a synthetic biology‐based platform to construct stable production cell lines, which ...
Yu‐Chieh Lin +5 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
Orphanet Journal of Rare Diseases, 2021 Background Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life.
Tom Burke +4 more
doaj +1 more source
Clinical Pharmacology &Therapeutics, EarlyView.All biotherapeutics have the potential to induce an immunogenic response and generate anti‐drug antibodies (ADAs), especially when administered as multiple doses over prolonged periods. However, a clinically meaningful effect of ADAs can be difficult to identify to communicate the impact of immunogenicity on drug exposure, safety and efficacy outcomes ...
Susan Richards +13 more
wiley +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
Impact of Model‐Informed Drug Development on Drug Development Cycle Times and Clinical Trial Cost
Clinical Pharmacology &Therapeutics, EarlyView.Model‐informed drug development (MIDD) integrates data to quantify benefit/risk informing objective drug discovery and development decisions. An additional critical benefit of MIDD is postulated to be improvement in trial and program efficiencies. While the application of MIDD has grown, there have been no clear examples across programs to demonstrate ...
Vaishali Sahasrabudhe +4 more
wiley +1 more source
Prenatal and molecular diagnosis of hemophilia B [PDF]
American Journal of Hematology, 1996 Prenatal diagnosis was carried out on a woman who had previously given birth to a son with a spontaneous mutation of C-->T transition at nt 31133 of the factor IX (F.IX) gene. The diagnosis was performed on chorionic villi sampling by the method of amplification-created restriction site (ACRS).
Jyh-Pyng Gau +3 more
openaire +3 more sources
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 +6 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
Сибирский научный медицинский журнал, 2019 Objective was to study genetic markers of thrombophilia in patients with hemophilia, which can affect the course of the disease and contribute to thrombotic complications. Material and methods.
T. Yu. Polyanskaya +11 more
doaj +1 more source