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
A rAAV2‐producing yeast screening model to identify host proteins enhancing rAAV DNA replication and vector yield
Sign Up to like & getrecommendations! Published in 2019 at "Biotechnology Progress"
DOI: 10.1002/btpr.2725
Abstract: Recombinant adeno‐associated viral vectors (rAAV) are promising therapies for genetic diseases. Although current platforms for recombinant vector production can generate drug material for pre‐clinical and clinical studies, rAAV biomanufacturing will eventually face commercial supply challenges… read more here.
Keywords: vector; dna replication; raav; yield ... 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
MRI-Guided Focused Ultrasound for Targeted Delivery of rAAV to the Brain.
Sign Up to like & getrecommendations! Published in 2019 at "Methods in molecular biology"
DOI: 10.1007/978-1-4939-9139-6_10
Abstract: Recombinant adeno-associated viral (rAAV) vectors are a promising tool for therapeutic gene delivery to the brain. However, the delivery of rAAVs across the blood-brain barrier (BBB) and entry into the brain remains a major challenge… read more here.
Keywords: focused ultrasound; mri guided; delivery; raav ... See more keywords
Impact of Inverted Terminal Repeat Integrity on rAAV8 Production Using the Baculovirus/Sf9 Cells System
Sign Up to like & getrecommendations! Published in 2017 at "Human Gene Therapy Methods"
DOI: 10.1089/hgtb.2016.133
Abstract: Adeno-associated virus (AAV) inverted terminal repeats (ITRs) are key elements of AAV. These guanine-cytosine-rich structures are involved in the replication and encapsidation of the AAV genome, along with its integration in and excision from the… read more here.
Keywords: system; production; dna; itr ... See more keywords
Accurate Identification and Quantification of DNA Species by Next-Generation Sequencing in Adeno-Associated Viral Vectors Produced in Insect Cells.
Sign Up to like & getrecommendations! Published in 2017 at "Human gene therapy methods"
DOI: 10.1089/hgtb.2016.185
Abstract: Recombinant adeno-associated viral (rAAV) vectors have proven excellent tools for the treatment of many genetic diseases and other complex diseases. However, the illegitimate encapsidation of DNA contaminants within viral particles constitutes a major safety concern… read more here.
Keywords: quantification dna; dna; insect cells; raav ... See more keywords
Manufacturing Clinical Grade Recombinant Adeno-Associated Virus Using Invertebrate Cell Lines.
Sign Up to like & getrecommendations! Published in 2017 at "Human gene therapy"
DOI: 10.1089/hum.2017.042
Abstract: Recombinant adeno-associated virus (rAAV) vectors are proving to be a reliable gene transfer system for several clinical applications, with an increasing body of evidence supporting safety and efficacy. Realizing the clinical and commercial potential of… read more here.
Keywords: invertebrate cell; recombinant adeno; associated virus; raav ... See more keywords
Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy?
Sign Up to like & getrecommendations! Published in 2017 at "Human gene therapy"
DOI: 10.1089/hum.2017.29039.trf
Abstract: AS MORE RECOMBINANT adeno-associated virus (rAAV) products are tested in or licensed for clinical use, the need for methods for large-scale, highquality manufacturing have increased proportionally. One of the recurring themes of recent advances in… read more here.
Keywords: product; purification; raav; adeno associated ... See more keywords
Development of a beta cell-specific expression control element for rAAV.
Sign Up to like & getrecommendations! Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2021.219
Abstract: Diabetes mellitus, caused by loss or dysfunction of the insulin producing beta cells of the pancreas, is a promising target for rAAV-mediated gene therapy. To target potential therapeutic payloads specifically to beta cells, a cell-type… read more here.
Keywords: beta; raav; expression; beta cell ... See more keywords
Titration of Recombinant Adeno-Associated Virus (rAAV) Genome Copy Number Using Real-Time Quantitative Polymerase Chain Reaction (qPCR).
Sign Up to like & getrecommendations! Published in 2020 at "Cold Spring Harbor protocols"
DOI: 10.1101/pdb.prot095646
Abstract: This protocol is used to determine the concentration of DNase-resistant vector genomes (i.e., packaged in the capsid) in purified recombinant adeno-associated virus (rAAV) preparations. The protocol begins with treatment of the vector stock with DNase… read more here.
Keywords: vector; dnase; real time; time ... See more keywords
rAAV expressing recombinant antibody for emergency prevention and long-term prophylaxis of COVID-19
Sign Up to like & getrecommendations! Published in 2023 at "Frontiers in Immunology"
DOI: 10.3389/fimmu.2023.1129245
Abstract: Introduction Numerous agents for prophylaxis of SARS-CoV-2-induced diseases are currently registered for the clinical use. Formation of the immunity happens within several weeks following vaccine administration which is their key disadvantage. In contrast, drugs based… read more here.
Keywords: emergency; long term; raav; prophylaxis ... See more keywords