Results 11 to 20 of about 36,553 (255)
Innate Immune Sensing of Adeno-Associated Virus Vectors [PDF]
Human Gene TherapyCox Terhorst +2 more
exaly +2 more sources
Molecular Therapy: Methods & Clinical Development, 2022 Gene therapy using neurotropic adeno-associated virus vectors represents an emerging solution for genetic disorders affecting the central nervous system. The first approved central nervous system-targeting adeno-associated virus gene therapy, Zolgensma®,
Makoto Horiuchi +10 more
doaj +1 more source
Adeno-Associated Virus Vectors and Hematology [PDF]
Blood, 1999 Gene therapists have a special fondness for hematology. Initially, this was due to the many inherited diseases that could potentially be cured by ex vivo genetic modification of hematopoietic stem cells. Once the early murine leukemia virus (MLV) vectors were found to transduce only a small percentage of primate stem cells, the search began for other ...
D W, Russell, M A, Kay
openaire +2 more sources
Infectious Entry Pathway of Adeno-Associated Virus and Adeno-Associated Virus Vectors [PDF]
Journal of Virology, 2000 ABSTRACT We have investigated the infectious entry pathway of adeno-associated virus (AAV) and recombinant AAV vectors by assessing AAV-mediated gene transfer and by covalently conjugating fluorophores to AAV and monitoring entry by fluorescence microscopy.
J S, Bartlett, R, Wilcher, R J, Samulski
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Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
Frontiers in Immunology, 2020 Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer. However, the high homology with the parental wild-type virus, which often
Giuseppe Ronzitti +2 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2022 Given the increasing number of gene transfer therapy studies either completed or underway, there is growing attention to the importance of preexisting adaptive immunity to the viral vectors used.
Jerry R. Mendell +7 more
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Membranes, 2022 One major challenge in the development of nanoparticle-based therapeutics, including viral vectors for the delivery of gene therapies, is the development of cost-effective purification technologies.
Liang-Kai Chu +3 more
doaj +1 more source
Frontiers in Immunology, 2021 Conventional vaccinations and immunotherapies have encountered major roadblocks in preventing infectious diseases like HIV, influenza, and malaria. These challenges are due to the high genomic variation and immunomodulatory mechanisms inherent to these ...
Wei Zhan +11 more
doaj +1 more source
Biosafety of Recombinant Adeno-associated Virus Vectors [PDF]
Current Gene Therapy, 2014 It is hoped that the use of gene transfer technology to treat both monogenetic and acquired diseases may soon become a common therapy option in medicine. For gene therapy to achieve this objective, any gene delivery method will have to meet several criteria, including ease of manufacturing, efficient gene transfer to target tissue, long-term gene ...
Dismuke, D.J. +2 more
openaire +3 more sources
Adenovirus as a Vector and Oncolytic Virus
Current Issues in Molecular Biology, 2023 Adenoviral vectors, both oncolytic viruses and gene delivery vectors, are among the earliest approved and commercialised vectors for gene therapy. Adenoviruses have high cytotoxicity and immunogenicity. Therefore, lentiviruses or adeno-associated viruses
Wataru Matsunaga, Akinobu Gotoh
doaj +1 more source