Recombination and Population Mosaic of a Multifunctional Viral Gene, Adeno-Associated Virus cap [PDF]
, 2008 Homologous recombination is a dominant force in evolution and results in genetic mosaics. To detect evidence of recombination events and assess the biological significance of genetic mosaics, genome sequences for various viral populations of reasonably ...
Danos, O, Myers, R, Takeuchi, Y
core +7 more sources
Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription
Scientific Reports, 2022 Duchenne muscular dystrophy is a severe neuromuscular disease causing a progressive muscle wasting due to mutations in the DMD gene that lead to the absence of dystrophin protein.
Amédée Mollard +11 more
doaj +1 more source
Adeno-associated Virus DNA Replication [PDF]
Cold Spring Harbor Symposia on Quantitative Biology, 1979 ABSTRACT. A model for the replication of adeno-associated virus (AAV) DNA is presented. The AAV genome is a linear single-stranded DNA molecule which contains a 145 nucleotide inverted terminal repetition of which the first 125 nucleotides form a palindromic sequence.
William W. Hauswirth +3 more
openaire +3 more sources
Progress in the use of adeno-associated viral vectors for gene therapy [PDF]
, 2004 The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core +1 more source
Viral Hybrid Vectors for Somatic Integration - Are They the Better Solution? [PDF]
, 2009 The turbulent history of clinical trials in viral gene therapy has taught us important lessons about vector design and safety issues. Much effort was spent on analyzing genotoxicity after somatic integration of therapeutic DNA into the host genome. Based
Ehrhardt, Anja +2 more
core +4 more sources
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
Baker, AH +4 more
core +2 more sources
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology.
Human Gene Therapy, 2020 The administration of adeno-associated virus (AAV) vectors to nonhuman primates (NHP) via the blood or cerebrospinal fluid (CSF) can lead to dorsal root ganglion (DRG) pathology.
J. Hordeaux +16 more
semanticscholar +1 more source
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
doaj +1 more source
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
G. Ronzitti, D. Gross, F. Mingozzi
semanticscholar +1 more source
Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]
, 2002 The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Davidoff, AM +12 more
core +1 more source