Article
Virology
Mario Mietzsch, Courtnee Eddington, Ariana Jose, Jane Hsi, Paul Chipman, Tom Henley, Modassir Choudhry, Robert McKenna, Mavis Agbandje-McKenna
Summary: Novel Rep hybrids from different AAV serotypes can significantly reduce the percentage of empty capsids in various AAV serotypes, while improving the yields of genome-containing AAV capsids. These Rep chimeras have the potential to revolutionize rAAV production and enhance the therapeutic efficacy of AAV-based gene therapies.
JOURNAL OF VIROLOGY
(2021)
Article
Multidisciplinary Sciences
Maximilian Hoerner, Carolina Jerez-Longres, Anna Hudek, Sebastian Hook, O. Sascha Yousefi, Wolfgang W. A. Schamel, Cindy Hoerner, Matias D. Zurbriggen, Haifeng Ye, Hanna J. Wagner, Wilfried Weber
Summary: The study developed an OptoAAV system that transfers genetic information into target cells upon red light illumination, enabling gene transfer with single-cell resolution, compatible with various cell lines and primary cells, and allowing spatially resolved transduction with different transgenes through sequential application of multiple OptoAAVs. This approach is likely extendable to other classes of viral vectors, facilitating advances in basic and applied genetic research.
Article
Virology
Junping Zhang, Xiangping Yu, Ping Guo, Jenni Firrman, Derek Pouchnik, Yong Diao, Richard Jude Samulski, Weidong Xiao
Summary: Historically, adeno-associated virus (AAV) defective interfering particles (DI) were considered abnormal virions resulting from replication and encapsidation errors. Through single virion genome analysis, it was revealed that a major category of DI particles contain double-stranded DNA genomes in snapback configuration. The 5'-SBGs and 3'-SBGs have different functions in regulating AAV gene expression levels in their dimer configurations.
Article
Biochemical Research Methods
Yingjun Ma, Junjiang Zhong
Summary: Viral infection involves a large number of protein-protein interactions (PPIs) between the virus and the host, and the identification of these PPIs plays an important role in revealing viral infection and pathogenesis. We propose a novel computational framework, LTDSSL, to determine human-virus PPIs under different disease types. Experimental results show that LTDSSL has better predictive performance for both new disease types and new triples than the state-of-the-art methods.
BRIEFINGS IN BIOINFORMATICS
(2023)
Review
Chemistry, Medicinal
Jessica Merjane, Roger Chung, Rickie Patani, Leszek Lisowski
Summary: Despite the mysterious etiology, differentiated treatments are required for ALS to address both familial and sporadic cases. Targeting mechanisms of defective protein homeostasis and RNA processing, as well as exploring the use of gene therapy through adeno-associated virus (AAV) for gene delivery to the CNS, might provide potential therapeutic interventions. Overall, there is a strong need for disease modifying treatments for ALS that can effectively treat the full spectrum of cases.
MEDICINAL RESEARCH REVIEWS
(2023)
Review
Medicine, General & Internal
Ekaterina M. Shitik, Igor K. Shalik, Dmitry V. Yudkin
Summary: Recombinant adeno-associated virus (rAAV) is a leading platform for gene delivery, but improvements are needed to enhance vector performance. This review focuses on modifying ITRs, transgene cassettes, and Rep proteins as ways to improve rAAV vectors and increase transduction efficiency.
FRONTIERS IN MEDICINE
(2023)
Article
Biotechnology & Applied Microbiology
Shantoshini Dash, David M. Sharon, Alaka Mullick, Amine A. Kamen
Summary: This study reveals that only a small proportion of cells are capable of producing measurable levels of assembled AAV capsids despite high transfection efficiencies and nominal vector yields. Increasing the proportion of productive cells could significantly increase vector titer. The flow cytometry assay used in this study may serve as a useful metric for optimizing transfection-based AAV vector manufacturing platforms.
BIOTECHNOLOGY AND BIOENGINEERING
(2022)
Article
Virology
Xiaohui Sun, Lianyi Zang, Xiaoying Liu, Shanshan Jiang, Xianping Zhang, Dan Zhao, Kaijie Shang, Tao Zhou, Changxiang Zhu, Xiaoping Zhu
Summary: The protein p27 of Tomato chlorosis virus (ToCV) plays a crucial role in the systemic infection of the virus and interacts with Solanum lycopersicum catalases (SlCAT). By directly inhibiting the anti-ToCV processes mediated by SlCAT1 or SlCAT2, p27 can promote viral infection.
Article
Biochemical Research Methods
Joseph M. Scarrott, Yusuf B. Johari, Thilo H. Pohle, Ping Liu, Ayda Mayer, David C. James
Summary: Researchers found that adding small molecule bioactive chemicals Nocodazole and M344 can significantly increase the production of rAAV virus by human embryonic kidney cells, up to three times higher than untreated cells.
BIOTECHNOLOGY JOURNAL
(2023)
Article
Virology
Franziska Tanneberger, Ahmed Abd El Wahed, Melina Fischer, Sandra Blome, Uwe Truyen
Summary: African swine fever poses a global threat to the pig industry and wild suids, with affected wild boar populations in Germany playing a significant role. The study found that MVAV can be used as a guide to select the best disinfectant concentration, with 0.1% peracetic acid effectively inactivating ASFV.
Article
Immunology
Julia R. Diller, Maximilian H. Carter, Yuta Kanai, Shania Sanchez, Takeshi Kobayashi, Kristen M. Ogden
Summary: Analysis of infant sera samples under different vaccination conditions revealed that vaccination with the multivalent RotaTeq vaccine can induce a broader neutralizing antibody response, and the addition of a dose of RotaTeq can also enhance the neutralizing response to the G type. In addition, there were differences in infectivity, replication, and neutralization of some monoreassortant viruses in different cell types.
JOURNAL OF INFECTIOUS DISEASES
(2021)
Article
Physics, Multidisciplinary
J. C. Phillips, Marcelo A. Moret, Gilney F. Zebende, Carson C. Chow
Summary: This study predicts the transmissibility and vaccine effectiveness of the novel coronavirus SARS-CoV-2 and its variants using a thermodynamic model of protein conformations. The findings suggest that the mutations in SARS-CoV-2 and its variants will not diminish the efficacy of spike-based vaccines and may even enhance viral infectivity.
PHYSICA A-STATISTICAL MECHANICS AND ITS APPLICATIONS
(2022)
Article
Biotechnology & Applied Microbiology
Hiroaki Oyama, Kentaro Ishii, Takahiro Maruno, Tetsuo Torisu, Susumu Uchiyama
Summary: Recombinant adeno-associated virus (AAV) is an important tool in human gene therapy, and this study identified two VP3-related components in AAV vectors using sodium dodecyl sulfate-polyacrylamide gel electrophoresis, capillary gel electrophoresis, and liquid chromatography-UV-mass spectrometry. The study suggests that complementary evaluation of VP components using CGE and LC-UV-MS methods is necessary for accurate quantification and understanding of VP stoichiometry in AAV vectors.
HUMAN GENE THERAPY
(2021)
Article
Biochemical Research Methods
Patrick C. Freitag, Fabian Brandl, Dominik Brucher, Fabian Weiss, Birgit Dreier, Andreas Pluckthun
Summary: Efficient and cell-specific DNA delivery is crucial for the safe and effective use of gene delivery technologies. The adapter technology described in this study redirects a commonly used human adenovirus to a specific cell population by blocking its natural tropism and allowing fusion with a retargeting module. By expanding the nature of the cell-binding portion, the modularity and applicability of this adapter technology is increased, allowing receptor-specific transduction mediated by different types of binding modules. This modular design enables efficient and specific mono- or dual-targeting without the need for tedious optimization procedures, making it valuable for gene therapy, synthetic biology, and biotechnology research.
BIOCONJUGATE CHEMISTRY
(2022)
Article
Biochemistry & Molecular Biology
Ranjha Khan, Babak Oskouian, Joanna Y. Lee, Jeffrey B. Hodgin, Yingbao Yang, Gizachew Tassew, Julie D. Saba
Summary: Sphingosine-1-phosphate lyase insufficiency syndrome (SPLIS) is a metabolic disorder caused by inactivating mutations in the SGPL1 gene. A study found that AAV-SPL gene therapy can prolong the survival of knockout mice and prevent or delay the onset of SPLIS phenotypes. The efficacy of a modified AAV-SPL 2.0, called AAV-SPL 2.0, was tested and showed equal or better results. However, improved kidney targeting may be necessary for optimal gene therapy.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)