Process Improvement of Adeno-Associated Virus Production
Frontiers in Chemical Engineering, 2022 Adeno-associated viruses (AAVs) have been well characterized and used to deliver therapeutic genes for diseases treatment in clinics and basic research. This study used the triple transient transfection of AAV-DJ/8 as a model expression system to develop
Jia-Shiung Guan +7 more
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Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]
, 2003 A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM +4 more
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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
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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
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Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]
, 2001 Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A +5 more
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Adeno-associated virus: fit to serve [PDF]
Current Opinion in Virology, 2014 Adeno-associated virus (AAV) is a helper-dependent parvovirus which has not been linked with human disease. This aspect, in combination with its broad cell and tissue tropism, and limited viral host response has made it an attractive vector system for gene therapy.
Eric Zinn, Luk H Vandenberghe
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Gene Therapy for Cardiovascular Disease [PDF]
, 2003 The last decade has seen substantial advances in the development of gene therapy strategies and vector technology for the treatment of a diverse number of diseases, with a view to translating the successes observed in animal models into the clinic ...
Baker, Andrew H. +3 more
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Perinatal Gene Transfer to the Liver [PDF]
, 2011 The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM +6 more
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Expression Profiles of Bovine Adeno-Associated Virus and Avian Adeno-Associated Virus Display Significant Similarity to That of Adeno-Associated Virus Type 5 [PDF]
Journal of Virology, 2006 ABSTRACT We present the first detailed expression profiles of nonprimate-derived adeno-associated viruses, namely, bovine adeno-associated virus (B-AAV) and avian adeno-associated virus (A-AAV), which were obtained after the infection of cell lines derived from their natural hosts.
Jianming, Qiu +2 more
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Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease
Blood Advances, 2017 : Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a target for genetic correction.
Randall K. Merling +14 more
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