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Industrial Biotechnology 2022

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  Scaling AAV Production: Easing the Transition from Laboratory Scales to Commercial Manufacturing Aden associated virus (AAV) has emerged as the leading vector for gene therapy delivery. Compared with options such as lent virus and adenovirus, AAV exhibits a strong safety profile because it has low pathogenicity and requires a helper virus to replicate. AAV is also capable of long-term gene expression, and it can infect both dividing and no dividing cells ( 1–5 ). Developers of advanced therapies have found such advantages to be quite attractive. As of January 2021, two gene therapy products have gained US Food and Drug Administration (FDA) approval: Luxturna (voretigene neparvovec) from Spark Therapeutics and Zolgensma (onasemnogene abeparvovec) from AveXis/Novartis. Both products use AAV vectors (serotypes 2 and 9, respectively), as do many candidate therapies moving through clinical studies. Cost-effective AAV manufacturing remains elusive, however. Yields and expression titers fro
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Molecular Biology Conference

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  A novel COVID-19 vaccine using modified bacterial DNA Experimental vaccine based on altered plasmid DNA produced antibody response in mice that effectively blocked cell infection across all variants of concern tested. Researchers describe a different way to build a COVID-19 vaccine, one that would, in theory, remain effective against new and emerging variants and could be taken as a pill, by inhalation or other delivery methods. Researchers at University of California San Diego School of Medicine, with colleagues elsewhere, describe a different way to build a COVID-19 vaccine, one that would, in theory, remain effective against new and emerging variants and could be taken as a pill, by inhalation or other delivery methods. Their findings publish in the July 21, 2022 online issue of  PLOS Pathogens . The research involved building plasmids genetically altered to contain bits of genetic material specifically intended to target a vulnerability in the SARS-CoV-2 virus's spi
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Industrial Biotechnology Conference 2022

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  Opportunities for Spaceflight to Advance Stem Cell Science, Biomanufacturing, and Disease Modeling The secret to producing large batches of stem cells more efficiently may lie in the near-zero gravity conditions of space. Scientists at Cedars-Sinai report that microgravity has the potential to contribute to life-saving advances on Earth by facilitating the rapid mass production of stem cells. A paper (“Biomanufacturing in low Earth orbit for regenerative medicine”), led by the Cedars Sinai team and published in  Stem Cell Reports , highlights key opportunities discussed during the 2020 Biomanufacturing in Space Symposium, to expand the manufacture of stem cells in space. Attendees at the virtual space symposium in December identified more than 50 potential commercial opportunities for conducting biomanufacturing work in space, according to the Cedars-Sinai paper. The most promising fell into three categories: disease modeling, biofabrication, and stem-cell–derived products. The newl
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Molecular Biology and Nucleic Acid 2022

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  Development of RNA G-quadruplex (rG4)-targeting L-RNA aptamers by rG4-SELEX Introduction : Guanine (G)-rich sequences of single-stranded DNA and RNA can fold into stable, intra- or intermolecular secondary structures called G-quadruplexes (dG4s and rG4s). These nucleic acid structure scaffolds are composed of stacks of G-quartets and can be further stabilized in the presence of monovalent ions, preferentially K +  or Na +  but not Li. Earlier findings have shown that G4s play important roles in various cellular events, including but not limited to DNA replication , DNA damage repair, transcription, translation, RNA metabolism and epigenetic remodelling. The ability to regulate fundamental biological processes, as well as their chemically interesting structures, makes G4s promising targets for potential cancer, antimicrobial and antiviral treatments. With the mounting interests in the biological role of G4s, more structure-specific, sensitive and low-cytotoxicity tools are needed to n
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Industrial Biotech Conference 2022

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                  Tips for Increasing Transfection Efficiency Chemical transfection , which relies on the formation of a condensed complex of positively charged  liposomal or non-liposomal  reagents and negatively charged nucleic acids, is the most common method for delivering foreign genetic material into cultured cells. Complexes are attracted to the negatively charged cell membrane and potentially pass through it via mechanisms involving endocytosis and phagocytosis. 1 “The negative to positive ratio impacts the complex size and the overall positive charge. The best ratio is cell type dependent,” says  Sandy Tseng, PhD,   technical support scientist, Mirus Bio. Chemical methods offer a slew of advantages: relatively low cytotoxicity, ease of use, cost effectiveness, low likelihood for unintended mutagenesis , no viral vector involvement, no discrimination of type of nucleic acid, and researcher safety. But the correct protocol needs to be identified and optimized to maximize transfe
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Nucleic acid 2022

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Overlap in pro-inflammatory genes and pathways between COVID-19 and MIS-C                                                                                                                                                                        In a recent study posted to the medrivix pre-print server, researchers in the United States characterized differential host immune responses in acute coronavirus disease 2019 (COVID-19) and  multisysteminflammatory syndrome in children  (MIS-C) to inform future development of novel biomarkers for both diseases.   Background To date, COVID-19 and MIS-C, both caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), have claimed more lives of children than pediatric mortality from influenza. Both these diseases manifest highly inflammatory states and have distinct signatures of cell injury and cell death, with more heterogeneity and multi-organ involvement observed in MIS-C. Further, both these diseases show different levels of e
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