Results 171 to 180 of about 73,809 (216)

Rapid multiplex liver gene-editing in mice using adeno-associated virus 8 or lipid nanoparticles. [PDF]

Mol Ther Adv
Wu D, Bolt I, Tolenaars DW, Duijst S, In Het Panhuis W, Hofstraat SRJ, Zwolsman R, van der Meel R, Paulusma CC, van de Graaf SFJ.   +9 more
europepmc   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Optimization of Tangential Flow Filtration for High-Yield, Scalable Downstream Processing of Adeno-Associated Virus. [PDF]

Membranes (Basel)
Cardoso S, Bollmann F, Tappe A.
europepmc   +1 more source

What Makes an “Ideal” Cell Line for Recombinant Adeno‐Associated Virus Production?

Biotechnology and Bioengineering, EarlyView.
Several host cell types have been used to produce rAAVs to date. Cell line‐specific traits that are beneficial in the context of rAAV manufacturing are reviewed here, with the goal of developing a consensus on the ‘ideal’ characteristics that an rAAV production host should possess (created with Biorender.com).
James Conheady, Nicholas Donohue, Alexandra Bogdanovic, Sharon Davin, Brian Glennon, Niall Barron, C. Mark Smales   +6 more
wiley   +1 more source

Generation and characterization of a HEK293 cell line optimized for recombinant adeno-associated virus production. [PDF]

Biotechnol Rep (Amst)
Vona M, Bodenmann I, Perrenoud MA, Buchs R, Yasli PK, Nanni L, Daveau R, Félix A, Stolte J, Hov AK, Chollet B, Schuepbach T, Ley D, Guzman E, Fisch I, Mermod N.   +15 more
europepmc   +1 more source

Purification of Advanced Therapeutics by Flow‐Through Pseudo‐Affinity Chromatography: Bi‐Specific mABs, Fc Fusion Proteins, and Adeno‐Associated Viruses

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Modern biopharmaceutical manufacturing requires purification platforms capable of processing structurally and functionally diverse products while addressing the challenge of removing persistent and high‐risk host cell proteins (HCPs).
Wenning Chu, Eduardo Barbieri, Arianna Minzoni, Shriarjun Shastry, Jacob P. LeBarre, Qingbo Shu, Taufika Williams, Xuanwen Li, Divya Chandra, Xuankuo Xu, Jerome Bill, David Roush, Joseph Taris, Patrick Gilbert, Andre Kiesewetter, Oliver Rammo, Michael Schulte, Christopher Major, Trina Mouchahoir, Steven Cramer, Cristiana Boi, Ruben G. Carbonell, Abraham Lenhoff, Stefano Menegatti   +23 more
wiley   +1 more source

The delivery challenge of adeno-associated virus vector-based gene therapies for neurological diseases. [PDF]

Front Neurosci
Pak A, Wear D, Tahmasian N, Min JY, Premraj D, Gibbs R, Spencer K, Fang S, Zerbes T, Krishnan M, Nasser Z, Schmitt-Ulms G.   +11 more
europepmc   +1 more source

High Cell Density Cultivation of HEK293 Cells Using a 2L Membrane‐Stirred Bioreactor

Biotechnology and Bioengineering, EarlyView.
ABSTRACT The use of perfusion systems for the continuous cultivation of animal cells at high cell density (HCD) for the production of recombinant proteins, viruses, and viral vectors has many advantages. Besides an enhancement of volumetric productivity and high cell viability, the product quality can be improved through a steady‐state environment ...
Lennart Jacobtorweihe, Yasemin van Heuvel, Kathrina Scheiermann, Patrick Bongartz, Yvonne Genzel, Udo Reichl   +5 more
wiley   +1 more source

Integrated computational and experimental immunoengineering of adeno-associated virus capsid T cell epitopes in mice. [PDF]

Nat Commun
Bing S, Eskandarian A, Smith S, Sajib AM, Warrington S, Saleh S, Najera SS, J D'Esposito R, Santana-Quintero L, Mazor R.   +9 more
europepmc   +1 more source

Enhancing Adeno‐Associated Virus 2 Capsid Engineering Through Co‐Evolutionary Coupling

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated virus (AAV) has emerged as a leading platform for gene therapy. However, to unlock the full potential, their manufacturing yields, stability, and efficacy must all be improved. Rational design is limited by poor predictability and the potential impact of mutations on multiple important vector properties.
Sirimar Laosinwattana, Yiwen Li, Braulio Carrillo Sanchez, Ayesha Khan, Paul A. Dalby   +4 more
wiley   +1 more source