Recombinant adeno-associated virus (AAV) is a valuable and often used gene therapy vector. With increased demand for highly purified virus comes the need for a standardized purification procedure that is applicable across many serotypes and includes bioengineered viruses. Currently cesium chloride banding or affinity chromatography are the predominate forms of purification. These approaches expose the final purified virus to toxic contaminants or are highly capsid dependent and may require significant optimization to isolate purified AAV. These methods may also limit crude viral lysate processing volume resulting in a significant loss of viral titer. To circumvent these issues, we have developed an AAV purification protocol independent of toxic compounds, supernatant volume and capsid moiety. This purification method standardizes virus purification across native serotype and bioengineered mosaic capsids.
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http://dx.doi.org/10.14440/jbm.2016.102 | DOI Listing |
Int J Mol Sci
January 2025
National Institute for Bioprocessing Research and Training, Foster Avenue, Mount Merrion, Blackrock, A94 X099 Dublin, Ireland.
Recombinant Adeno-associated virus (rAAV) is a popular vector for treating genetic diseases caused by absent or defective genes. rAAVs can be produced that contain a therapeutic transgene, i.e.
View Article and Find Full Text PDFGenes (Basel)
January 2025
School of Pharmacy, Center of Graduate Studies, West Coast University, 590 N Vermont Ave, Los Angeles, CA 90004, USA.
Gene therapy has emerged as a promising frontier in the management of diabetes, offering innovative approaches to address both type 1 and type 2 diabetes. This narrative review examines the advancements in gene therapy applications, focusing on both animal and human studies, and includes a total of 11 studies in adherence to PRISMA guidelines. These studies utilize various viral vectors, such as adeno-associated virus (AAV) and lentivirus, to deliver genes that regulate insulin production and enhance angiogenesis.
View Article and Find Full Text PDFMol Med
January 2025
Division of Gastroenterology, The First Affiliated Hospital, Sun Yat-Sen University, Guangzhou, P. R. China.
Background: Obesity is a significant risk factor for severe acute pancreatitis (SAP) and is typically associated with increased intestinal permeability. Understanding the role of specific molecules can help reduce the risk of developing SAP. Claudin 11 (CLDN11), a member of the Claudin family, regulates the permeability of various internal barriers.
View Article and Find Full Text PDFCurr Pharm Biotechnol
January 2025
College of Pharmaceutical Science, Zhejiang University of Technology, Hangzhou 310000, China.
Background: Gene therapy has been effectively applied in many biological studies and for the treatment of many genetic or cancer diseases. Currently, Recombinant Adeno- Associated Viruses (rAAVs) are one of the main types of delivery vectors used for gene therapy. rAAV vectors produced via the Sf9 cells have the advantages of high rAAV yields, easy scaleup, and low cost.
View Article and Find Full Text PDFExp Eye Res
January 2025
Institutes of Biology and Medical Sciences, Soochow University, Suzhou, 215000, China; Key Laboratory of Geriatric Diseases and Immunology, Ministry of Education, Institutes of Biology and Medical Sciences, Suzhou Medical College of Soochow University, Suzhou, 215123, China. Electronic address:
Due to its unique physiological structure and functions, the eye has received considerable attention in the field of adeno-associated virus (AAV) gene therapy. Inherited retinal degenerative diseases, which arise from pathogenic mutations in mRNA transcripts expressed in the eye's photoreceptor cells or retinal pigment epithelium (RPE), are the most common cause of vision loss. However, current retinal gene therapy mostly involves subretinal injection of therapeutic genes, which treats a limited area, entails retinal detachment, and requires sophisticated techniques.
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