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Bioscience

Gene delivery systems

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Displaying 1 - 10 of 10

Interlaboratory Measurement of Adeno-Associated Virus: Comparative Quantification of Full and Empty Capsids

December 26, 2024
Author(s)
Sean Lehman, Wyatt Vreeland, Jerome Jacques, Sharee Adams-Hall, Shreya Ahuja, Adnan Arnaout, Hunter Balduf, Ivan Budyak, Thomas Cleveland, Dennis Delgado, James Deng, Brandon Doyle, David Duewer, Carsten Elger, Jeffrey Fagan, Tim Guo, Jorge Haller, Luisa Hilgenfeld, Van Hoang, Allison Huldin, Matthew Hyatt, Sambit Kar, Sandeep Kedia, Bashkim Kokona, Amy Liu, Li Ma, Diane McCarthy, He Meng, Easton Noble, Veronika Oettle, Andrew Pla, Thomas Powers, Dean Ripple, Herbert Runnels, Raphael Ruppert, Florian Semmelmann, Christopher Sims, Saurabh Singh, Ronald Toth, Austin Vogt, Sabine Wenzel, Neal Whitaker, Zhiwen Yang, Brandon Zhuang
Recombinant adeno-associated virus (AAV) is one of the main viral vector-based gene therapy platforms. AAV is a virus consisting of a ≈25 nm diameter capsid with a ≈4.7 kb cargo capacity. The safety and efficacy depend on the correct encapsidation of the

Standards and Metrology for Viral Vectors as Molecular Tools: Outcomes from a CCQM Workshop

May 24, 2024
Author(s)
Janathan Campbell, Neil Almond, Y Bae, Ravneet Bhuller, Andrea Briones, S-Y CHO, Megan Cleveland, Thomas Cleveland, Francis Galaway, Hua-Jun He, U Herbrand, Jim Huggett, Sarah Kempster, Ibolya Kepiro, Afifa Khan, Edward Kwee, Wilson Li, Sheng Lin-Gibson, Luise Luckau, Caterina Minelli, M Ryadnov, I Searing, Lili Wang, Alexandra Whale, Julian Braybrook
Viral vectors are agents enabling gene transfer and genome editing and have widespread utility across the healthcare and biotechnology sectors. In January 2023, the International Bureau for Weights and Measures' Consultative Committee for Amount of

Quantification of mRNA in Lipid Nanoparticles Using Mass Spectrometry

December 26, 2023
Author(s)
Mark Lowenthal, Abigail Antonishek, Karen Phinney
Lipid nanoparticle-encapsulated messenger RNA (LNP-mRNA) holds great promise as a novel modality for treating a broad range of diseases. The ability to quantify mRNA accurately in therapeutic products helps to ensure consistency and safety. Here we

Quantitation and integrity evaluation of RNA genome in lentiviral vectors by direct reverse transcription-droplet digital PCR (direct RT-ddPCR)

September 2, 2023
Author(s)
Zhiyong He, Edward Kwee, Megan Cleveland, Kenneth Cole, Sheng Lin-Gibson, Hua-Jun He
Abstract Lentiviral vectors (LV) have proven to be powerful tools for stable gene delivery in both dividing and non-dividing cells. Approval of these LVs for use in clinical applications has been achieved by improvements in LV design. Critically important

Reference standards for accurate validation and optimization of assays that determine integrated Lentiviral vector copy number in transduced cells

July 11, 2021
Author(s)
Barbara S. Paugh, Lajos Baranyi, Andre Roy, Hua-Jun He, Lindsay Harris, Kenneth Cole, Caroline Raimund, Patricia S. Langan, Moria Artlip, Srikanta Jana, Rimas J. Orentas, Sheng Lin-Gibson, Winfried Krueger, Boro Dropuli?
Lentiviral vectors (LV) have emerged as a robust technology for therapeutic gene delivery into human cells as advanced medicinal products. As these products are increasingly commercialized, there are concomitant demands for their characterization to ensure

Measurement and Standardization Challenges for Exosome-Based Delivery Vectors

September 3, 2020
Author(s)
Bryant Nelson, Hao-Wei Wang, Samantha Maragh, Paul DeRose, Elzafir B. Elsheikh, Wyatt Vreeland, Ionita Ghiran, Jennifer Jones
Extracellular vesicles (EVs), and in particular exosomes, have the potential to revolutionize the development and efficient delivery of clinical therapeutics. In this Perspective, we focus on providing a brief introduction to the landscape of exosome-based

The effect of protein corona composition on the interaction of carbon nanotubes with human blood platelets

July 31, 2014
Author(s)
Silvia H. De Paoli Lacerda, Lukas Diduch, Tseday Tegegn, Martina Orecna, Michael Strader, Elena Karnaukhova, John Bonevich, Abdu Alayash, Karel Holada, Jan Simak
Carbon nanotubes (CNT) are one of the most promising nanomaterials for use in medicine. Applications of CNT in drug/gene delivery, diagnostics and tissue engineering, among other applications, require contact of CNT with blood. Therefore, evaluation of

Characterization of Linear and Branched Polyethylenimine (PEI) by ESI-MS and MALDI-TOF-MS

December 31, 2002
Author(s)
Kathleen M. Flynn, M Konaklieva, J P. Gu, Charles M. Guttman, S Wetzel
Polyethylenimine (PEI) is being used as a DNA transfection agent. Complexes of DNA and PEI are known to pass through the wall of a cell, and studies have reported that PEI s efficiency as a gene delivery vehicle depends on its molecular weight. Molecular
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