Resolving hidden subpopulations of filled AAVs by probing capsid integrity

IF 4.6 2区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Molecular Therapy-Methods & Clinical Development Pub Date : 2024-08-29 DOI:10.1016/j.omtm.2024.101322
Weida Wu, Georgios Katsikis
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通过探测囊壳完整性解析填充型 AAV 隐藏亚群
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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". Comparison and cross-validation of long-read and short-read target-enrichment sequencing methods to assess AAV vector integration into host genome. Toward a translational gene therapy for mucolipidosis IV. Identification of a novel neutralization epitope in rhesus AAVs. AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection.
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