Articles with "raav" as a keyword



Unveiling Small Non‐Coding RNA Dynamics During Recombinant Adeno‐Associated Virus Production

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Published in 2025 at "Biotechnology Journal"

DOI: 10.1002/biot.70092

Abstract: Recombinant adeno‐associated viruses (rAAVs) play a pivotal role in gene therapy, yet the molecular interactions underlying rAAV production in host cells remain incompletely understood. Non‐coding RNAs (ncRNAs), particularly microRNAs (miRNAs) and small nucleolar RNAs (snoRNAs),… read more here.

Keywords: recombinant adeno; adeno associated; non coding; raav ... See more keywords

Critical challenges and advances in recombinant adeno-associated virus (rAAV) biomanufacturing.

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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

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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

Scalable process development for rAAV transient transfection production using computational fluid dynamics modeling

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Published in 2025 at "Biotechnology Progress"

DOI: 10.1002/btpr.70028

Abstract: Recombinant adeno‐associated virus (rAAV) is a promising delivery vehicle for cell and gene therapies. Upstream development faces challenges like low productivity and inconsistent performance despite advancements. This study presents a scale‐up design for robust rAAV… read more here.

Keywords: raav; process; culture; cell ... See more keywords
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A Quantitative PCR Screening Method for Adeno-Associated Viral Vector 2-mediated Gene Doping.

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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.

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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

Use of cesium chloride density gradient ultracentrifugation for the purification and characterization of recombinant adeno-associated virus

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Published in 2025 at "European Biophysics Journal"

DOI: 10.1007/s00249-025-01751-1

Abstract: Recombinant adeno-associated virus (rAAV) has been widely used as an effective delivery tool in gene therapy. One of the challenges facing the production of high-quality rAAV is optimization of the production and purification methodologies. Cesium… read more here.

Keywords: raav; density gradient; purification; cscl dguc ... See more keywords

Crucial aspects for maintaining rAAV stability

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Published in 2024 at "Scientific Reports"

DOI: 10.1038/s41598-024-79369-0

Abstract: The storage of rAAV vectors for gene therapy applications is critical for ensuring a constant product quality and defined amount of medication at the time of administration. Therefore, we determined the influence of different storage… read more here.

Keywords: raav; raav stability; raav9 agal; biopotency ... See more keywords

Impact of Inverted Terminal Repeat Integrity on rAAV8 Production Using the Baculovirus/Sf9 Cells System

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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.

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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.

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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