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Order Bunyavirales
Published in Paul Pumpens, Peter Pushko, Philippe Le Mercier, Virus-Like Particles, 2022
Paul Pumpens, Peter Pushko, Philippe Le Mercier
An example of the pseudotyping approach was represented by preparation of a recombinant virus from La Crosse (LACV) and Jamestown Canyon (JCV) viruses, both belonging to the California encephalitis virus serogroup of orthobunyaviruses (Bennett et al. 2012). As a result, LACV expressing the attachment/fusion glycoproteins of JCV demonstrated protection in mice against lethal challenge with both viruses and against challenge with JCV in rhesus monkeys (Bennett et al. 2012).
Gene Therapy and Small Molecules Used in the Treatment of Cystic Fibrosis
Published in Yashwant Pathak, Gene Delivery, 2022
Manish P. Patel, Uma G. Daryai, Mansi N. Athalye, Praful D. Bharadia, Jayvadan Patel
Other viruses used are Human (HIV), simian (SIV) and feline (FIV) immunodeficiency virus, as well as equine infectious anemia virus (EIAV). Vector platforms are being developed for cystic fibrosis gene therapy. As these vectors do not have a natural tropism for the lung, pseudotyping with appropriate envelope proteins is required to achieve efficient transduction. The vesicular stomatitis virus G (VSVG) protein, which is widely used, does not transduce airway epithelium efficiently via the apical membrane, but the transduction efficiency of vesicular stomatitis virus G can be increased by pre-administration of the tight junction openers that allow access of the virus to the basolateral membrane (Griesenbach, Pytel, and Alton, 2015).
Gene Transfer into Human Hematopoietic Stem Cells
Published in Richard K. Burt, Alberto M. Marmont, Stem Cell Therapy for Autoimmune Disease, 2019
Serguei Kisselev, Tatiana Seregina, Richard K. Burt, Charles J. Link
Another viral envelope is derived from vesicular stomatitis virus glycoprotein (VSV-G). VSV-G pseudotyping has been employed as another family of pseudotyped retroviral vector systems. VSV-G pseudotyped murine retroviral vectors possess a number of advantages. The VSV-G envelope is more stable and permits vector concentration without significant loss of activity.60 Furthermore, VSV-G envelope possesses a tropism to some phospholipids (phosphadidylserine, phosphatidylinositol and GM3 ganglioside) and enters into target cells via endocytic pathway.61 Therefore, HSC do not require expression of a receptor protein for absorption and intracellular transportation of VSV-G pseudotyped vectors and could be transduced easily.62 The main disadvantage of VSV-G envelope is its direct toxicity when expressed during production of pseudotyped vectors in VPC.63
A tool with many applications: vesicular stomatitis virus in research and medicine
Published in Expert Opinion on Biological Therapy, 2020
Altar M. Munis, Emma M. Bentley, Yasuhiro Takeuchi
Current research focuses on improving four aspects of oncolytic VSV variants: resistance to host antiviral responses, enhanced onco-selectivity, improved oncolysis, and safer vectors. In addition to the M protein mutant mentioned above, several other M protein variants (e.g. A1 and A2) have been generated in the hope of targeting IFN-1 sensitive tumors [195,204]. In addition, heterologous pseudotyping strategies have been used to try to decrease the neurotropism of the virus (e.g. using envelope proteins from Sindbis virus [205–207], Lassa virus [208], measles virus [209]) and to avoid the humoral immune response generated against VSV.G (e.g. by using the nonimmunogenic LCMV envelope [210]). Another measure taken to increase the safety profile and onco-selectivity of the virus was the incorporation of surface targeting markers (e.g. anti-her2/neu receptor [205,207] and microRNA targets [211]).
An update on gene therapy for lysosomal storage disorders
Published in Expert Opinion on Biological Therapy, 2019
Murtaza S. Nagree, Simone Scalia, William M. McKillop, Jeffrey A. Medin
AAV is also being used as a systemically administered gene therapy vector. Much as with LV envelope pseudotypes, AAV tissue specificity results from capsid serotypes that often transduce multiple cell types efficiently [62]. For example, systemic injection of AAV9 serotyped vector leads to transduction of brain parenchymal cells, suggesting blood-brain barrier (BBB) crossover [63]. AAV8 serotyped vector can transduce hepatocytes in the liver very efficiently [64]. AAV2 serotyped vector has a broader tropism [64]. Pseudotyping, combining the capsid and genome from different viral serotypes, can be used to further alter tropism. For example, AAV2/8, a vector containing the genome of AAV2 packaged with the capsid from AAV8, has been used in treatment models for Pompe disease [13]. In a major distinction between AAV and LV, once recombinant AAV enters a cell it predominantly resides episomally [15]. While target cells that divide less frequently retain AAV episomes long term, quickly dividing cells may require repeated AAV administration. AAVs are thus promising vectors for the treatment of LSDs with primarily neuronal or muscle-based manifestations.
Advancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques
Published in Expert Opinion on Biological Therapy, 2022
Kyle D Kovacs, Thomas A Ciulla, Szilárd Kiss
This is the most commonly employed gene delivery system within ophthalmology, particularly recombinant AAVs that consist of defective single-strand DNA parvoviruses. They have been popularly, and successfully, used for retinal indications due to the non-replicating, nonintegrating (episomal) features, low immunogenicity, and ability to transduce nondividing cells [69]. They are less likely to initiate an immunologic response compared with adenoviral or lentiviral delivery. However, a significant drawback to rAAVs is their capacity (~4.5–5.0 kb), limiting their use to specific disease targets. There are 13 different naturally occurring AAVs (AAV1-AAV13) isolated from primates. Further refinement of particular constructs can be achieved via pseudotyping or cross-packing, in which different serotypes and capsids can be utilized. For example, a vector like AAV2/8 can be generated consisting of an AAV2 serotype within an AAV8 capsid [70]. Dual AAV vectors have also been developed which increase the capacity of this delivery system [71]. Some therapies incorporate both of these concepts, such as the dual AAV2/8 vectors delivered concurrently in the treatment of autosomal dominant RHO-associated RP. In this therapy, AAV2/8 vectors held either a Cas9 expression cassette or guide RNAs targeting the genomic DNA up and downstream of the RHO codon and a wild-type RHO expression cassette [72]. Dual AAVs deliver separate gene fragments within independent AAVs and are being utilized for CRISPR-Cas9 approaches (see corresponding section), though there remain concerns over low efficiency in this approach [73–75].