DNA Minicircle Technology Improves Purity of Adeno-associated Viral Vector Preparations
نویسندگان
چکیده
منابع مشابه
DNA Minicircle Technology Improves Purity of Adeno-associated Viral Vector Preparations
Adeno-associated viral (AAV) vectors are considered as one of the most promising delivery systems in human gene therapy. In addition, AAV vectors are frequently applied tools in preclinical and basic research. Despite this success, manufacturing pure AAV vector preparations remains a difficult task. While empty capsids can be removed from vector preparations owing to their lower density, state-...
متن کاملIncreasing the minicircle DNA purity using an enhanced triplex DNA technology to eliminate DNA contaminants
DNA vectors for human gene therapy have to meet the efficacy and safety requirements. Minicircles (MCs), a class of optimized DNA vectors free of plasmid backbone (PB) DNAs, have emerged as promising candidates because of their superior transgene expression profiles. However, the existence of impure DNAs, including the unrecombined MC producing plasmid (PP) and PB circle, in the MC products mad...
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Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...
متن کاملAdeno-associated viral vector transduction of human mesenchymal stem cells.
Mesenchymal stem cells (MSCs) have received considerable attention in the emerging field of regenerative medicine. One aspect of MSC research focuses on genetically modifying the cells with the aim of enhancing their regenerative potential. Adeno-associated virus (AAV) holds promise as a vector for human gene therapy, primarily due to its lack of pathogenicity and low risk of insertional mutage...
متن کامل6 Adeno - Associated Viral Vectors
Adeno-associated virus (AAV) is a non-pathogenic humanDNAviruswith a unique profile of biological properties that have been of interest to molecular virologists for many years (Berns, 1990; Carter, 1990; Carter et al., 1990). Recently,AAV has also attracted interest as a vector for gene transfer (Carter, 1992; Flotte, 1993a; Hermonat andMuzyczka, 1984; Tratschin et al., 1984). In a general sens...
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ژورنال
عنوان ژورنال: Molecular Therapy - Nucleic Acids
سال: 2016
ISSN: 2162-2531
DOI: 10.1038/mtna.2016.60