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Pharmacology of Recombinant Adeno-associated Virus Production [PDF]
Molecular Therapy: Methods & Clinical Development, 2018 Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases.
Magalie Penaud-Budloo +3 more
doaj +5 more sources
Frontiers in Microbiology, 2023 Adeno-associated viruses (AAV) are among the foremost vectors for in vivo gene therapy. A number of monoclonal antibodies against several serotypes of AAV have previously been prepared.
Edward E. Large, Michael S. Chapman
doaj +3 more sources
Protein Carrier Adeno-Associated Virus. [PDF]
ACS NanoAdeno-associated virus (AAV) has emerged as a leading platform for gene therapy, enabling the delivery of therapeutic DNA to target cells. However, the potential of AAV to deliver protein payloads has been unexplored. In this study, we engineered a protein carrier AAV (pcAAV) to package and deliver proteins by inserting binding domains on the interior ...
Hoffmann MD +4 more
europepmc +3 more sources
Genetics of adeno-associated virus: isolation and preliminary characterization of adeno-associated virus type 2 mutants [PDF]
Journal of Virology, 1984 We constructed insertion and deletion mutants with mutations within the adeno-associated virus (AAV) sequences of the infectious recombinant plasmid pSM620. Studies of these mutants revealed at least three AAV phenotypes. Mutants with mutations between 11 and 42 map units were partially or completely defective for rescue and replication of the AAV ...
Paul L. Hermonat +4 more
openalex +4 more sources
Retinal Penetrating Adeno-Associated Virus. [PDF]
Invest Ophthalmol Vis SciThe most common method of delivery of genes to the outer retina uses recombinant adeno-associated virus (AAV) injected into the subretinal space using a surgical procedure. In contrast, most drugs are delivered to the retina using an intravitreal approach in an office setting. The objective of the current study was to develop AAV vectors that can reach
Kumar B +3 more
europepmc +3 more sources
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 +3 more sources
Adeno-Associated Virus (AAV) Versus Immune Response
Viruses, 2019 Decades ago, Friedmann and Roblin postulated several barriers to gene therapy, including tissue targeting, delivery across the blood⁻brain barrier (BBB), and host immune responses. These issues remain pertinent till today.
Joseph Rabinowitz +2 more
doaj +2 more sources
Virus Binding and Internalization Assay for Adeno-associated Virus
Bio-Protocol, 2017 The binding and internalization of adeno-associated virus (AAV) is an important determinant of viral infectivity and tropism. The ability to dissect these two tightly connected cellular processes would allow better understanding and provide insight on ...
Garrett Berry, Victor Tse
doaj +5 more sources
An essential receptor for adeno-associated virus infection [PDF]
Nature, 2015 Adeno-associated virus (AAV) vectors are currently the leading candidates for virus-based gene therapies because of their broad tissue tropism, non-pathogenic nature and low immunogenicity.
Sirika Pillay +11 more
semanticscholar +4 more sources
Effectiveness of resveratrol in inducing adeno-associated virus as a potential definitive therapy for SCN5A mutation in Brugada syndrome: a narrative review [PDF]
The Egyptian Heart JournalObjectives Brugada syndrome (BrS) is a hereditary channelopathy that affects cardiac electrical signal transmission, with SCN5A gene mutation being the most common cause.
Andin Zahrani Pateda +3 more
doaj +2 more sources