Results 31 to 40 of about 9,370,992 (242)
Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats [PDF]
, 2008 Background: Vectors based on adeno-associated virus-8 (AAV8) have shown efficiency and efficacy for liver-directed gene therapy protocols following intravascular injection, particularly in relation to haemophilia gene therapy. AAV8 has also been proposed
Amit Nathwani +10 more
core +1 more source
Molecular Therapy: Methods & Clinical Development, 2019 Targeting specific cell types in the mammalian inner ear is important for treating genetic hearing loss due to the different cell type-specific functions.
Min-A Kim +7 more
doaj +1 more source
Green Analytical Chemistry, 2022 Herein we describe a platform method to study the charge heterogeneity of adeno-associated virus (AAV) capsid proteins. The method, based on imaged capillary isoelectric focusing (icIEF) with native fluorescence imaging detection, was validated, and can ...
Jiaqi Wu, Chris Heger
doaj +1 more source
Optimization of Large‐Scale Adeno‐Associated Virus (AAV) Production
Current Protocols, 2023 Abstract Genetic manipulation in vivo is a critical method for mechanistically understanding gene function in disease and physiological processes. To facilitate this, embryonic transgenesis in popular animal models like mice has been developed.
Alina S, Bilal +6 more
openaire +2 more sources
Viruses, 2021 Adeno-associated virus (AAV) was first characterized as small “defective” contaminant particles in a simian adenovirus preparation in 1965. Since then, a recombinant platform of AAV (rAAV) has become one of the leading candidates for gene therapy ...
Jacquelyn J. Bower +3 more
doaj +1 more source
Development of Therapies for Spinal Muscular Atrophy Using Gene Therapy and Nanotechnology [PDF]
, 2013 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
The state of the art of adeno-associated virus-based vectors in gene therapy
Virology Journal, 2007 The adeno-associated virus (AAV) has rapidly gained popularity in gene therapy since the establishment of the first AAV2 infectious clone, in 1982, due to some of their distinguishing characteristics such as lack of pathogenicity, wide range of ...
Nardi Nance, Coura Renata
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2018 Adeno-associated virus (AAV) has provided the gene therapy field with the most powerful in vivo gene delivery vector to realize safe, efficacious, and sustainable therapeutic gene expression.
Dan Wang +14 more
doaj +1 more source
Nature Communications, 2020 Replacing mutant genes with wildtype copies using adeno-associated virus (AAV) has been explored for the treatment of inherited retinopathies, but the low cargo limit restricts its use.
Koji M. Nishiguchi +4 more
doaj +1 more source
Identification of adeno-associated virus contamination in cell and virus stocks by PCR
BioTechniques, 2004 To further understand the biology of adeno-associated virus (AAV) and identify the presence of AAV in laboratory samples, we have developed a sensitive PCR-based assay using degenerate primers based on the sequence of seven diverse AAV isolates.
Hisako Katano +3 more
doaj +1 more source