Molecular Signature of Astrocytes for Gene Delivery by the Synthetic Adeno-Associated Viral Vector rAAV9P1.
Sign Up to like & getrecommendations! Published in 2022 at "Advanced science"
DOI: 10.1002/advs.202104979
Abstract: Astrocytes have crucial functions in the central nervous system (CNS) and are major players in many CNS diseases. Research on astrocyte-centered diseases requires efficient and well-characterized gene transfer vectors. Vectors derived from the Adeno-associated virus… read more here.
Keywords: adeno associated; raav9p1; gene; human astrocytes ... See more keywords
Increased recombinant adeno‐associated virus production by HEK293 cells using small molecule chemical additives
Sign Up to like & getrecommendations! Published in 2022 at "Biotechnology Journal"
DOI: 10.1002/biot.202200450
Abstract: Recombinant adeno‐associated virus (rAAV) has established itself as a highly efficacious gene delivery vector with a well characterised safety profile allowing broad clinical application. Recent successes in rAAV‐mediated gene therapy clinical trials will continue to… read more here.
Keywords: small molecule; recombinant adeno; associated virus; adeno associated ... See more keywords
Process economics evaluation and optimization of adeno-associated virus downstream processing.
Sign Up to like & getrecommendations! Published in 2023 at "Biotechnology and bioengineering"
DOI: 10.1002/bit.28402
Abstract: Adeno-associated virus (AAV) manufacturing has traditionally focused upon lab-scale techniques to culture and purify vector products, leading to limitations in production capacity. The tool presented in this paper assesses the feasibility of using non-scalable technologies… read more here.
Keywords: economics; process economics; associated virus; adeno associated ... See more keywords
Critical challenges and advances in recombinant adeno-associated virus (rAAV) biomanufacturing.
Sign Up to like & getrecommendations! Published in 2023 at "Biotechnology and bioengineering"
DOI: 10.1002/bit.28412
Abstract: Gene therapy is a promising therapeutic approach for genetic and acquired diseases nowadays. Among DNA delivery vectors, recombinant adeno-associated virus (rAAV) is one of the most effective and safest vectors used in commercial drugs and… read more here.
Keywords: raav; recombinant adeno; associated virus; adeno associated ... See more keywords
Enrichment of adeno-associated virus serotype 5 full capsids by anion exchange chromatography with dual salt elution gradients.
Sign Up to like & getrecommendations! Published in 2023 at "Biotechnology and bioengineering"
DOI: 10.1002/bit.28453
Abstract: Adeno-associated virus-based gene therapies have demonstrated substantial therapeutic benefit for the treatment of genetic disorders. In manufacturing processes, viral capsids are produced with and without the encapsidated gene of interest. Capsids devoid of the gene… read more here.
Keywords: associated virus; adeno associated; full capsids; anion exchange ... See more keywords
Transcriptomic features reveal molecular signatures associated with recombinant adeno-associated virus production in HEK293 cells.
Sign Up to like & getrecommendations! Published in 2023 at "Biotechnology progress"
DOI: 10.1002/btpr.3346
Abstract: The development of gene therapies based on recombinant adeno-associated viruses (rAAVs) has grown exponentially, so the current rAAV manufacturing platform needs to be more efficient to satisfy rising demands. Viral production exerts great demand on… read more here.
Keywords: transcriptomic features; recombinant adeno; adeno associated; viral production ... See more keywords
A Quantitative PCR Screening Method for Adeno-Associated Viral Vector 2-mediated Gene Doping.
Sign Up to like & getrecommendations! Published in 2021 at "Drug testing and analysis"
DOI: 10.1002/dta.3152
Abstract: Gene therapy is currently prohibited in human and equine athletes and novel analytical methods are needed for its detection. Most in vivo products use non-integrating, recombinant viral vectors derived from adeno-associated virus (AAV) to deliver… read more here.
Keywords: quantitative pcr; gene doping; raav; adeno associated ... See more keywords
Liver Directed Adeno-Associated Viral Vectors to Treat Metabolic Disease.
Sign Up to like & getrecommendations! Published in 2023 at "Journal of inherited metabolic disease"
DOI: 10.1002/jimd.12637
Abstract: The liver is the metabolic center of the body and an ideal target for gene therapy of inherited metabolic disorders (IMDs). Adeno-associated viral (AAV) vectors can deliver transgenes to the liver with high efficiency and… read more here.
Keywords: adeno associated; gene therapy; metabolic disease; liver directed ... See more keywords
Age‐stratified adeno‐associated virus serotype 3 neutralizing and total antibody prevalence in hemophilia A patients from India
Sign Up to like & getrecommendations! Published in 2022 at "Journal of Medical Virology"
DOI: 10.1002/jmv.27859
Abstract: Gene therapy using an adeno‐associated virus (AAV) vector offers a new treatment option for individuals with monogenetic disorders. The major bottleneck is the presence of pre‐existing anti‐AAV antibodies, which impacts its use. Even very low… read more here.
Keywords: hemophilia patients; associated virus; adeno associated; age ... See more keywords
Extracellular vesicles: nature's nanoparticles for improving gene transfer with adeno-associated virus vectors.
Sign Up to like & getrecommendations! Published in 2018 at "Wiley interdisciplinary reviews. Nanomedicine and nanobiotechnology"
DOI: 10.1002/wnan.1488
Abstract: Gene therapy, the ability to treat a disease at the level of nucleic acid, has journeyed from science fiction, to hard lessons learned from early clinical trials, to improved technologies with efficacy in patients for… read more here.
Keywords: associated virus; aav vectors; adeno associated; gene transfer ... See more keywords
In Vivo Delivery of Cassettes Encoding Anti-HBV Primary MicroRNAs Using an Ancestral Adeno-Associated Viral Vector.
Sign Up to like & getrecommendations! Published in 2020 at "Methods in molecular biology"
DOI: 10.1007/978-1-0716-0290-4_10
Abstract: Chronic hepatitis B, a liver disease resulting from persisting hepatitis B virus (HBV) infection, remains a global health challenge despite the availability of an effective vaccine. Various preclinical studies using adeno-associated viruses (AAVs) to deliver… read more here.
Keywords: anti hbv; encoding anti; cassettes encoding; delivery cassettes ... See more keywords