Results 11 to 20 of about 9,649,206 (293)
Novel Cytotoxic Vectors Based on Adeno-Associated Virus [PDF]
Vectors based on adeno-associated virus (AAV) are promising tools for gene therapy. The production of strongly toxic vectors, for example for cancer-directed gene transfer, is often unfeasible due to uncontrolled expression of toxic genes in vector ...
Johannes Kohlschütter +2 more
doaj +3 more sources
Biosafety of Recombinant Adeno-associated Virus Vectors [PDF]
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 +6 more sources
Fluorescence Microscopy in Adeno-Associated Virus Research
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 +2 more sources
Adeno-Associated Virus Vectors and Hematology [PDF]
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]
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
openaire +2 more sources
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
doaj +1 more source
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
Development of Therapies for Spinal Muscular Atrophy Using Gene Therapy and Nanotechnology [PDF]
Spinal muscular atrophy (SMA) is a genetic disease which is characterized by muscle weakness and atrophy. The disease arises from mutations in the survival motor neuron 1 (SMN1) gene causing degeneration of spinal cord motor neurons.
Little, Daniel
core +5 more sources
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
Adenovirus as a Vector and Oncolytic Virus
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

