用于基因治疗的现有抗体检测:考虑患者选择的临床临界值和辅助诊断要求

IF 4.6 2区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Molecular Therapy-Methods & Clinical Development Pub Date : 2024-02-20 DOI:10.1016/j.omtm.2024.101217
Manuela Braun, Claudia Lange, Philipp Schatz, Brian Long, Johannes Stanta, Boris Gorovits, Edit Tarcsa, Vibha Jawa, Tong-Yuan Yang, Wibke Lembke, Nicole Miller, Fraser McBlane, Louis Christodoulou, Daisy Yuill, Mark Milton
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Preexisting antibody assays for gene therapy: Considerations on clinical cut-off for patient selection and companion diagnostic requirements
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来源期刊
Molecular Therapy-Methods & Clinical Development
Molecular Therapy-Methods & Clinical Development Biochemistry, Genetics and Molecular Biology-Molecular Biology
CiteScore
9.90
自引率
4.30%
发文量
163
审稿时长
12 weeks
期刊介绍: The aim of Molecular Therapy—Methods & Clinical Development is to build upon the success of Molecular Therapy in publishing important peer-reviewed methods and procedures, as well as translational advances in the broad array of fields under the molecular therapy umbrella. Topics of particular interest within the journal''s scope include: Gene vector engineering and production, Methods for targeted genome editing and engineering, Methods and technology development for cell reprogramming and directed differentiation of pluripotent cells, Methods for gene and cell vector delivery, Development of biomaterials and nanoparticles for applications in gene and cell therapy and regenerative medicine, Analysis of gene and cell vector biodistribution and tracking, Pharmacology/toxicology studies of new and next-generation vectors, Methods for cell isolation, engineering, culture, expansion, and transplantation, Cell processing, storage, and banking for therapeutic application, Preclinical and QC/QA assay development, Translational and clinical scale-up and Good Manufacturing procedures and process development, Clinical protocol development, Computational and bioinformatic methods for analysis, modeling, or visualization of biological data, Negotiating the regulatory approval process and obtaining such approval for clinical trials.
期刊最新文献
What's in a word? Defining "gene therapy medicines". FastAd: A versatile toolkit for rapid generation of single adenoviruses or diverse adenoviral vector libraries. Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association. Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells. Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders.
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