Results 21 to 30 of about 36,553 (255)
Potential for cellular stress response to hepatic factor VIII expression from AAV vector
Molecular Therapy: Methods & Clinical Development, 2016 Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively.
Irene Zolotukhin +5 more
doaj +1 more source
JACC: Basic to Translational Science, 2019 Summary: Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients.
Stephen C. Kolwicz, Jr., PhD +7 more
doaj +1 more source
Adeno-associated Virus as a Mammalian DNA Vector [PDF]
Microbiology Spectrum, 2015 ABSTRACT In the nearly five decades since its accidental discovery, adeno-associated virus (AAV) has emerged as a highly versatile vector system for both research and clinical applications. A broad range of natural serotypes, as well as an increasing number of capsid variants, has combined to produce a repertoire of vectors with different ...
Max, Salganik +2 more
openaire +2 more sources
Innate Immune Response to Viral Vectors in Gene Therapy
Viruses, 2023 Viral vectors play a pivotal role in the field of gene therapy, with several related drugs having already gained clinical approval from the EMA and FDA.
Yixuan Wang, Wenwei Shao
doaj +1 more source
Neuroscience Insights, 2019 Recombinant adeno-associated virus (rAAV) vectors have emerged as the safe vehicles of choice for long-term gene transfer in mammalian nervous system. Recombinant adeno-associated virus–mediated localized gene transfer in adult nervous system following ...
Asad Jan +4 more
doaj +1 more source
A simplified purification protocol for recombinant adeno-associated virus vectors
Molecular Therapy: Methods & Clinical Development, 2014 We describe a new rapid, low cost, and scalable method for purification of various recombinant adeno-associated viruses (rAAVs) from the lysates of producer cells of either mammalian or insect origin. The method takes advantage of two general biochemical
Mark Potter +10 more
doaj +1 more source
Thermostability and in vivo performance of AAV9 in a film matrix
Communications Medicine, 2022 Doan et al. characterize and assess the stability of film matrix embedded Adeno-Associated Virus 9 (AAV9) vectors during storage and transport at ambient temperatures.
Trang Nguyen Kieu Doan +10 more
doaj +1 more source
Fluorescence Microscopy in Adeno-Associated Virus Research
Viruses, 2023 Research on adeno-associated virus (AAV) and its recombinant vectors as well as on fluorescence microscopy imaging is rapidly progressing driven by clinical applications and new technologies, respectively.
Susanne K. Golm +2 more
doaj +1 more source
Receptor targeting of adeno-associated virus vectors [PDF]
Gene Therapy, 2003 Adeno-associated virus (AAV) is a promising vector for human somatic gene therapy. However, its broad host range is a disadvantage for in vivo gene therapy, because it does not allow the selective tissue- or organ-restricted transduction required to enhance the safety and efficiency of the gene transfer.
H, Büning +6 more
openaire +2 more sources
Nature Communications, 2020 Adeno-associated virus is the basis of many gene therapies and gene transfer vectors. Here the authors report a pipeline to enable side-by-side comparison of pre-selected capsids in a high throughput manner.
Jonas Weinmann +17 more
doaj +1 more source