Results 51 to 60 of about 2,604,218 (267)

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, Han‐Jung Kuo, Min Lu, Thomas Mahl, George Aslanidi, Wei‐Shou Hu   +5 more
wiley   +1 more source

IQ Survey Results on Current Industry Practices: Part 2—Quantitative Evaluations of Immunogenicity Assessment

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, Insa Winzenborg, Doreen Luedtke, Tao Niu, Lora Hamuro, Karey Kowalski, Jocelyn H. Leu, Jianning Yang, Vaishnavi Ganti, Indranil Bhattacharya, Ivelina Gueorguieva, Nael Mostafa, Christine Grimaldi, Benjamin Wu   +13 more
wiley   +1 more source

The BM@N spectrometer at the NICA accelerator complex [PDF]

arXiv, 2023
BM@N (Baryonic Matter at Nuclotron) is the first experiment operating and taking data at the Nuclotron/NICA ion-accelerating complex.The aim of the BM@N experiment is to study interactions of relativistic heavy-ion beams with fixed targets. We present a technical description of the BM@N spectrometer including all its subsystems.
arxiv  

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]

, 2003
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM, Nathwani, AC, Ng, CYC, Spence, Y, Zhou, J   +4 more
core   +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, Timothy Nicholas, Gianluca Nucci, Cynthia J Musante, Brian Corrigan   +4 more
wiley   +1 more source

ORAL TREATMENT OF HEMOPHILIA [PDF]

, 2007
Disclosed herein is a simple method for the treatment of antigen-deficiency diseases, by orally administering to a subject a therapeutically effective amount of the deficient antigen, wherein the antigen is not present in a liposome.
Alpan, Oral, Kamala, Tirumalai, Matzinger, Polly, Velander, William Hugold   +3 more
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

Venous Thromboembolism Prophylaxis in Hemophilic Patients Undergoing Total Hip or Knee Arthroplasty: Insights from a Single-Center Experience

Medicina
Background and Objectives: Total hip replacement and total knee arthroplasty carry a high risk of postoperative venous thromboembolism (VTE); therefore, anticoagulation prophylaxis is recommended in these patients.
Oana-Viola Badulescu, Paul-Dan Sirbu, Manuela Ciocoiu, Maria Cristina Vladeanu, Carmen Elena Plesoianu, Andrei Bojan, Dan Iliescu-Halitchi, Razvan Tudor, Bogdan Huzum, Mihnea-Theodor Sirbu, Norin Forna, Gheorghe Sofron, Wilhelm Friedl, Iris Bararu-Bojan   +13 more
doaj   +1 more source

Combined Point of Care Tools Are Able to Improve Treatment Adherence and Health-Related Quality of Life in Patients with Severe Hemophilia: An Observational Prospective Study [PDF]

, 2019
Introduction: Ultrasound (US) assessment of joints is an evolving point of care tool for the detection of early joint arthropathy (Napolitano M, Kessler CM. Hemophilia A and B.
Di Minno, Giovanni, Di Minno, Matteo Nicola Dario, Mancuso, Salvatrice, Mansueto, Maria Francesca, Mauro, Giulia Letizia, Napolitano, Mariasanta, Raso, Simona, Scaturro, Dalila, Siragusa, Sergio   +8 more
core   +1 more source

Exploring the Potential and Advancements of Circular RNA Therapeutics

Exploration, EarlyView.
Given the remarkable advantages in terms of stability, sustained expression profile, safety, wide range of druggable targets, scalable and cost‐effective manufacturing capabilities, circRNA is currently undergoing intensive investigation for various therapeutic applications such as vaccines, protein replacement, genetic disease treatment, gene therapy,
Lei Wang, Lianqing Wang, Chunbo Dong, Jialong Liu, Guoxin Cui, Shan Gao, Zhida Liu   +6 more
wiley   +1 more source