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Journal articles on the topic 'AAV recombinant vector'

Author: Grafiati

Published: 25 January 2025

Last updated: 31 July 2025

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1

Wang, Xu-Shan, Benjawan Khuntirat, Keyun Qing, et al. "Characterization of Wild-Type Adeno-Associated Virus Type 2-Like Particles Generated during Recombinant Viral Vector Production and Strategies for Their Elimination." Journal of Virology 72, no. 7 (1998): 5472–80. http://dx.doi.org/10.1128/jvi.72.7.5472-5480.1998.

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ABSTRACT The pSub201-pAAV/Ad plasmid cotransfection system was developed to eliminate homologous recombination which leads to generation of the wild-type (wt) adeno-associated virus type 2 (AAV) during recombinant vector production. The extent of contamination with wt AAV has been documented to range between 0.01 and 10%. However, the precise mechanism of generation of the contaminating wt AAV remains unclear. To characterize the wt AAV genomes, recombinant viral stocks were used to infect human 293 cells in the presence of adenovirus. Southern blot analyses of viral replicative DNA intermedia

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2

Tejero, Marcos, Ozgun F. Duzenli, Colin Caine, Hisae Kuoch, and George Aslanidi. "Bioengineered Hybrid Rep 2/6 Gene Improves Encapsulation of a Single-Stranded Expression Cassette into AAV6 Vectors." Genes 14, no. 10 (2023): 1866. http://dx.doi.org/10.3390/genes14101866.

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The production of clinical-grade recombinant adeno-associated viral (AAV) vectors for gene therapy trials remains a major hurdle in the further advancement of the gene therapy field. During the past decades, AAV research has been predominantly focused on the development of new capsid modifications, vector-associated immunogenicity, and the scale-up vector production. However, limited studies have examined the possibility to manipulate non-structural components of AAV such as the Rep genes. Historically, naturally isolated, or recombinant library-derived AAV capsids have been produced using the

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3

Favaro, Patricia, Harre D. Downey, Federico Mingozzi, et al. "Safety of Recombinant Adeno-Associated Viral Vectors in a Large Animal Model." Blood 110, no. 11 (2007): 2586. http://dx.doi.org/10.1182/blood.v110.11.2586.2586.

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Abstract Recombinant adeno-associated viral (AAV) vector is a promising gene-based strategy for the treatment of several inherited diseases. Using AAV serotype 2 (AAV-2), the most common tested vector in humans, we have determined that the risk of germline transmission and the immune responses to both transgene product and/or vector-capsid proteins are critical obstacles to the safety of this strategy (Nat Med12:342, 2006). Recently, novel and more potent serotypes have emerged such as AAV-8 that allows efficient liver transduction following peripheral intravenous injection (IV). The major det

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4

Zhang, Huang-Ge, Jinfu Xie, Igor Dmitriev, et al. "Addition of Six-His-Tagged Peptide to the C Terminus of Adeno-Associated Virus VP3 Does Not Affect Viral Tropism or Production." Journal of Virology 76, no. 23 (2002): 12023–31. http://dx.doi.org/10.1128/jvi.76.23.12023-12031.2002.

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ABSTRACT Production of large quantities of recombinant adeno-associated virus (AAV) is difficult and not cost-effective. To overcome this problem, we have explored the feasibility of creating a recombinant AAV encoding a 6×His tag on the VP3 capsid protein. We generated a plasmid vector containing a six-His (6×His)-tagged AAV VP3. A second plasmid vector was generated that contained the full-length AAV capsid capable of producing VP1 and VP2, but not VP3 due to a mutation at position 2809 that encodes the start codon for VP3. These plasmids, necessary for production of AAV, were transfected in

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5

Grimm, Dirk, Kusum Pandey, Hiroyuki Nakai, Theresa A. Storm, and Mark A. Kay. "Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype." Journal of Virology 80, no. 1 (2006): 426–39. http://dx.doi.org/10.1128/jvi.80.1.426-439.2006.

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ABSTRACT We and others have recently reported highly efficient liver gene transfer with adeno-associated virus 8 (AAV-8) pseudotypes, i.e., AAV-2 genomes packaged into AAV-8 capsids. Here we studied whether liver transduction could be further enhanced by using viral DNA packaging sequences (inverted terminal repeats [ITRs]) derived from AAV genotypes other than 2. To this end, we generated two sets of vector constructs carrying expression cassettes embedding a gfp gene or the human factor IX (hfIX) gene flanked by ITRs from AAV genotypes 1 through 6. Initial in vitro analyses of gfp vector DNA

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6

Wright, J. Fraser. "Coating of AAV Vectors with Human Albumin Blocks Antibody Binding and Enables Transduction of Human Hepatocytes in the Presence of Neutralizing Antibodies." Blood 112, no. 11 (2008): 3542. http://dx.doi.org/10.1182/blood.v112.11.3542.3542.

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Abstract Neutralization of recombinant adeno-associated virus (AAV) gene transfer vectors by pre-existing antibodies is a significant barrier to clinical gene transfer using systemic administration. In a recent clinical trial for hemophilia B, while efficient gene transfer and therapeutic levels of hFIX were achieved in a human subject with low pre-existing antibodies to AAV2, no gene transfer was observed in a subject with a modest pre-existing AAV2 antibody titer of 1:17 who received the same dose (Manno et al. Nature Med.2006;12:342–347). With the objective to achieve consistent and efficie

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7

Fischer, Kyle B., Hannah K. Collins, and Edward M. Callaway. "Sources of off-target expression from recombinase-dependent AAV vectors and mitigation with cross-over insensitive ATG-out vectors." Proceedings of the National Academy of Sciences 116, no. 52 (2019): 27001–10. http://dx.doi.org/10.1073/pnas.1915974116.

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In combination with transgenic mouse lines expressing Cre or Flp recombinases in defined cell types, recombinase-dependent adeno-associated viruses (AAVs) have become the tool of choice for localized cell-type-targeted gene expression. Unfortunately, applications of this technique when expressing highly sensitive transgenes are impeded by off-target, or “leak” expression, from recombinase-dependent AAVs. We investigated this phenomenon and find that leak expression is mediated by both infrequent transcription from the inverted transgene in recombinant-dependent AAV designs and recombination ev

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8

Weger, Stefan. "High-Level rAAV Vector Production by rAdV-Mediated Amplification of Small Amounts of Input Vector." Viruses 15, no. 1 (2022): 64. http://dx.doi.org/10.3390/v15010064.

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The successful application of recombinant adeno-associated virus (rAAV) vectors for long-term transgene expression in clinical studies requires scalable production methods with genetically stable components. Due to their simple production scheme and the high viral titers achievable, first generation recombinant adenoviruses (rAdV) have long been taken into consideration as suitable tools for simultaneously providing both the helper functions and the AAV rep and cap genes for rAAV packaging. So far, however, such rAdV-rep/cap vectors have been difficult to generate and often turned out to be ge

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9

Hewitt, F. Curtis, Chengwen Li, Steven J. Gray, Shelley Cockrell, Michael Washburn, and R. Jude Samulski. "Reducing the Risk of Adeno-Associated Virus (AAV) Vector Mobilization with AAV Type 5 Vectors." Journal of Virology 83, no. 8 (2009): 3919–29. http://dx.doi.org/10.1128/jvi.02466-08.

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ABSTRACT Current adeno-associated virus (AAV) gene therapy vectors package a transgene flanked by the terminal repeats (TRs) of AAV type 2 (AAV2). Although these vectors are replication deficient, wild-type (wt) AAV2 prevalent in the human population could lead to replication and packaging of a type 2 TR (TR2)-flanked transgene in trans during superinfection by a helper virus, leading to “mobilization” of the vector genome from treated cells. More importantly, it appears likely that the majority of currently characterized AAV serotypes as well as the majority of new novel isolates are capable

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10

Lieber, André, Dirk S. Steinwaerder, Cheryl A. Carlson, and Mark A. Kay. "Integrating Adenovirus–Adeno-Associated Virus Hybrid Vectors Devoid of All Viral Genes." Journal of Virology 73, no. 11 (1999): 9314–24. http://dx.doi.org/10.1128/jvi.73.11.9314-9324.1999.

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ABSTRACT Recently, we demonstrated that inverted repeat sequences inserted into first-generation adenovirus (Ad) vector genomes mediate precise genomic rearrangements resulting in vector genomes devoid of all viral genes that are efficiently packaged into functional Ad capsids. As a specific application of this finding, we generated adenovirus–adeno-associated virus (AAV) hybrid vectors, first-generation Ad vectors containing AAV inverted terminal repeat sequences (ITRs) flanking a reporter gene cassette inserted into the E1 region. We hypothesized that the AAV ITRs present within the hybrid v

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11

Lebkowski, J. S., M. M. McNally, T. B. Okarma, and L. B. Lerch. "Adeno-associated virus: a vector system for efficient introduction and integration of DNA into a variety of mammalian cell types." Molecular and Cellular Biology 8, no. 10 (1988): 3988–96. http://dx.doi.org/10.1128/mcb.8.10.3988-3996.1988.

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Adeno-associated virus (AAV) is a single-stranded DNA parvovirus that is dependent on adenovirus or herpesvirus for reproductive functions. We describe the construction of recombinant AAV vectors containing the chloramphenicol acetyltransferase gene or the neomycin phosphotransferase gene. These vectors carried their respective genes into a wide variety of cell types, including primary skin fibroblasts and hematopoietic cells. Infection efficiencies varied with cell type and ranged up to 3.0%. Coinfection of two different recombinant viruses was also used to introduce two different sequences s

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12

Lebkowski, J. S., M. M. McNally, T. B. Okarma, and L. B. Lerch. "Adeno-associated virus: a vector system for efficient introduction and integration of DNA into a variety of mammalian cell types." Molecular and Cellular Biology 8, no. 10 (1988): 3988–96. http://dx.doi.org/10.1128/mcb.8.10.3988.

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Adeno-associated virus (AAV) is a single-stranded DNA parvovirus that is dependent on adenovirus or herpesvirus for reproductive functions. We describe the construction of recombinant AAV vectors containing the chloramphenicol acetyltransferase gene or the neomycin phosphotransferase gene. These vectors carried their respective genes into a wide variety of cell types, including primary skin fibroblasts and hematopoietic cells. Infection efficiencies varied with cell type and ranged up to 3.0%. Coinfection of two different recombinant viruses was also used to introduce two different sequences s

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13

Hauck, Bernd, Wei Zhao, Katherine High, and Weidong Xiao. "Intracellular Viral Processing, Not Single-Stranded DNA Accumulation, Is Crucial for Recombinant Adeno-Associated Virus Transduction." Journal of Virology 78, no. 24 (2004): 13678–86. http://dx.doi.org/10.1128/jvi.78.24.13678-13686.2004.

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ABSTRACT Adeno-associated virus (AAV) is a unique gene transfer vector which takes approximately 4 to 6 weeks to reach its expression plateau. The mechanism for this slow-rise expression profile was proposed to be inefficient second-strand DNA synthesis from the input single-stranded (ss) DNA viral genome. In order to clarify the status of ss AAV genomes, we generated AAV vectors labeled with bromodeoxyuridine (BrdU), a nucleotide analog that can be incorporated into the AAV genome and packaged into infectious virions. Since BrdU-DNA can be detected only by an anti-BrdU antibody when DNA is in

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14

Sollerbrant, Kerstin, Joacim Elmén, Claes Wahlestedt, et al. "A novel method using baculovirus-mediated gene transfer for production of recombinant adeno-associated virus vectors." Journal of General Virology 82, no. 9 (2001): 2051–60. http://dx.doi.org/10.1099/0022-1317-82-9-2051.

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The baculovirus Autographa californica multiple nucleopolyhedrosis virus causes non-productive infection in mammalian cells. Recombinant baculovirus therefore has the capability to transfer and express heterologous genes in these cells if a mammalian promoter governs the gene of interest. We have investigated the possibility of using baculovirus as a tool to produce recombinant adeno-associated virus (rAAV). AAV has become increasingly popular as a vector for gene therapy and functional genomics efforts, although its use is hampered by the lack of a simple and efficient vector production metho

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15

Mendoza, Skyler D., Yasmine El-Shamayleh, and Gregory D. Horwitz. "AAV-mediated delivery of optogenetic constructs to the macaque brain triggers humoral immune responses." Journal of Neurophysiology 117, no. 5 (2017): 2004–13. http://dx.doi.org/10.1152/jn.00780.2016.

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Gene delivery to the primate central nervous system via recombinant adeno-associated viral vectors (AAV) allows neurophysiologists to control and observe neural activity precisely. A current limitation of this approach is variability in vector transduction efficiency. Low levels of transduction can foil experimental manipulations, prompting vector readministration. The ability to make multiple vector injections into the same animal, even in cases where successful vector transduction has already been achieved, is also desirable. However, vector readministration has consequences for humoral immu

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16

Xiao, Xiao, Juan Li, Yeou-Ping Tsao, Devin Dressman, Eric P. Hoffman, and Jon F. Watchko. "Full Functional Rescue of a Complete Muscle (TA) in Dystrophic Hamsters by Adeno-Associated Virus Vector-Directed Gene Therapy." Journal of Virology 74, no. 3 (2000): 1436–42. http://dx.doi.org/10.1128/jvi.74.3.1436-1442.2000.

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ABSTRACT Limb girdle muscular dystrophy (LGMD) 2F is caused by mutations in the δ-sarcoglycan (SG) gene. Previously, we have shown successful application of a recombinant adeno-associated virus (AAV) vector for genetic and biochemical rescue in the Bio14.6 hamster, a homologous animal model for LGMD 2F (J. Li et al., Gene Ther. 6:74–82, 1999). In this report, we show efficient and long-term δ-SG expression accompanied by nearly complete recovery of physiological function deficits after a single-dose AAV vector injection into the tibialis anterior muscle of the dystrophic hamsters. AAV vector t

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17

Philpott, Nicola J., Catherine Giraud-Wali, Carolyn Dupuis, et al. "Efficient Integration of Recombinant Adeno-Associated Virus DNA Vectors Requires a p5-rep Sequence in cis." Journal of Virology 76, no. 11 (2002): 5411–21. http://dx.doi.org/10.1128/jvi.76.11.5411-5421.2002.

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ABSTRACT The initial aim of this study was to combine attributes of adeno-associated virus (AAV) and adenovirus (Ad) gene therapy vectors to generate an Ad-AAV hybrid vector allowing efficient site-specific integration with Ad vectors. In executing our experimental strategy, we found that, in addition to the known incompatibility of Rep expression and Ad growth, an equally large obstacle was presented by the inefficiency of the integration event when using traditional recombinant AAV (rAAV) vectors. This study has addressed both of these problems. We have shown that a first-generation Ad can b

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18

Nakai, Hiroyuki, Roland W. Herzog, J. Nathan Hagstrom, et al. "Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse Liver." Blood 91, no. 12 (1998): 4600–4607. http://dx.doi.org/10.1182/blood.v91.12.4600.412k22_4600_4607.

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Recombinant adeno-associated virus vectors (AAV) were prepared in high titer (1012 to 1013 particles/mL) for the expression of human factor IX after in vivo transduction of murine hepatocytes. Injection of AAV-CMV-F.IX (expression from the human cytomegalovirus IE enhancer/promoter) into the portal vein of adult mice resulted in no detectable human factor IX in plasma, but in mice injected intravenously as newborns with the same vector, expression was initially 55 to 110 ng/mL. The expression in the liver was mostly transient, and plasma levels decreased to undetectable levels within 5 weeks.

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19

Cao, Lei, Yuhong Liu, Matthew J. During, and Weidong Xiao. "High-Titer, Wild-Type Free Recombinant Adeno-Associated Virus Vector Production Using Intron-Containing Helper Plasmids." Journal of Virology 74, no. 24 (2000): 11456–63. http://dx.doi.org/10.1128/jvi.74.24.11456-11463.2000.

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ABSTRACT Recombinant adeno-associated virus (rAAV) is capable of directing long-term, high-level transgene expression without destructive cell-mediated immune responses. However, traditional packaging methods for rAAV vectors are generally inefficient and contaminated with replication-competent AAV (rcAAV) particles. Although wild-type AAV is not associated with any known human diseases, contaminating rcAAV particles may affect rAAV gene expression and are an uncontrolled variable in many AAV gene transfer studies. In the current study, a novel strategy was designed to both optimize AAV rep ge

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20

Domenger, Claire, and Dirk Grimm. "Next-generation AAV vectors—do not judge a virus (only) by its cover." Human Molecular Genetics 28, R1 (2019): R3—R14. http://dx.doi.org/10.1093/hmg/ddz148.

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AbstractRecombinant adeno-associated viruses (AAV) are under intensive investigation in numerous clinical trials after they have emerged as a highly promising vector for human gene therapy. Best exemplifying their power and potential is the authorization of three gene therapy products based on wild-type AAV serotypes, comprising Glybera (AAV1), Luxturna (AAV2) and, most recently, Zolgensma (AAV9). Nonetheless, it has also become evident that the current AAV vector generation will require improvements in transduction potency, antibody evasion and cell/tissue specificity to allow the use of lowe

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Ponnazhagan, Selvarangan, Gandham Mahendra, Sanjay Kumar, John A. Thompson, and Mark Castillas,. "Conjugate-Based Targeting of Recombinant Adeno-Associated Virus Type 2 Vectors by Using Avidin-Linked Ligands." Journal of Virology 76, no. 24 (2002): 12900–12907. http://dx.doi.org/10.1128/jvi.76.24.12900-12907.2002.

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ABSTRACT The development of targeted vectors, capable of tissue-specific transduction, remains one of the important aspects of vector modification for gene therapy applications. Recombinant adeno-associated virus type 2 (rAAV-2)-based vectors are nonpathogenic, have relatively low immunogenicity, and are capable of long-term transgene expression. AAV-2 vectors bind primarily to heparan sulfate proteoglycan (HSPG), a receptor that is present in many tissues and cell types. Because of the widespread expression of HSPG on many tissues, targeted transduction in vivo appears to be limited with AAV-

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22

Chatterjee, Saswati, Wei Li, Christie Ann Wong, et al. "Transduction of Primitive Human Marrow and Cord Blood-Derived Hematopoietic Progenitor Cells With Adeno-Associated Virus Vectors." Blood 93, no. 6 (1999): 1882–94. http://dx.doi.org/10.1182/blood.v93.6.1882.406k03_1882_1894.

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We evaluated the capacity of adeno-associated virus (AAV) vectors to transduce primitive human myeloid progenitor cells derived from marrow and cord blood in long-term cultures and long-term culture-initiating cell (LTC-IC) assays. Single-colony analyses showed that AAV vectors transduced CD34+ and CD34+38− clonogenic cells in long-term culture. Gene transfer was readily observed in LTC-ICs derived from 5-, 8-, and 10-week cultures. Recombinant AAV (rAAV) transduction was observed in every donor analyzed, although a wide range of gene transfer frequencies (5% to 100%) was noted. AAV transducti

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23

Duan, Dongsheng, Prerna Sharma, Jusan Yang, et al. "Circular Intermediates of Recombinant Adeno-Associated Virus Have Defined Structural Characteristics Responsible for Long-Term Episomal Persistence in Muscle Tissue." Journal of Virology 72, no. 11 (1998): 8568–77. http://dx.doi.org/10.1128/jvi.72.11.8568-8577.1998.

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ABSTRACT Adeno-associated viral (AAV) vectors have demonstrated great utility for long-term gene expression in muscle tissue. However, the mechanisms by which recombinant AAV (rAAV) genomes persist in muscle tissue remain unclear. Using a recombinant shuttle vector, we have demonstrated that circularized rAAV intermediates impart episomal persistence to rAAV genomes in muscle tissue. The majority of circular intermediates had a consistent head-to-tail configuration consisting of monomer genomes which slowly converted to large multimers of >12 kbp by 80 days postinfection. Importantly, long-

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24

Booth, M. J., A. Mistry, X. Li, A. Thrasher, and R. S. Coffin. "Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids." Gene Therapy 11, no. 10 (2004): 829–37. http://dx.doi.org/10.1038/sj.gt.3302226.

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25

Nakai, Hiroyuki, Roland W. Herzog, J. Nathan Hagstrom, et al. "Adeno-Associated Viral Vector-Mediated Gene Transfer of Human Blood Coagulation Factor IX Into Mouse Liver." Blood 91, no. 12 (1998): 4600–4607. http://dx.doi.org/10.1182/blood.v91.12.4600.

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Abstract Recombinant adeno-associated virus vectors (AAV) were prepared in high titer (1012 to 1013 particles/mL) for the expression of human factor IX after in vivo transduction of murine hepatocytes. Injection of AAV-CMV-F.IX (expression from the human cytomegalovirus IE enhancer/promoter) into the portal vein of adult mice resulted in no detectable human factor IX in plasma, but in mice injected intravenously as newborns with the same vector, expression was initially 55 to 110 ng/mL. The expression in the liver was mostly transient, and plasma levels decreased to undetectable levels within

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26

Ponnazhagan, Selvarangan, Kirsten A. Weigel, Sudhanshu P. Raikwar, Pinku Mukherjee, Mervin C. Yoder, and Arun Srivastava. "Recombinant Human Parvovirus B19 Vectors: Erythroid Cell-Specific Delivery and Expression of Transduced Genes." Journal of Virology 72, no. 6 (1998): 5224–30. http://dx.doi.org/10.1128/jvi.72.6.5224-5230.1998.

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ABSTRACT A novel packaging strategy combining the salient features of two human parvoviruses, namely the pathogenic parvovirus B19 and the nonpathogenic adeno-associated virus type 2 (AAV), was developed to achieve erythroid cell-specific delivery as well as expression of the transduced gene. The development of such a chimeric vector system was accomplished by packaging heterologous DNA sequences cloned within the inverted terminal repeats of AAV and subsequently packaging the DNA inside the capsid structure of B19 virus. Recombinant B19 virus particles were assembled, as evidenced by electron

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Duan, Dongsheng, Qiang Li, Aimee W. Kao, Yongping Yue, Jeffrey E. Pessin, and John F. Engelhardt. "Dynamin Is Required for Recombinant Adeno-Associated Virus Type 2 Infection." Journal of Virology 73, no. 12 (1999): 10371–76. http://dx.doi.org/10.1128/jvi.73.12.10371-10376.1999.

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ABSTRACT Recombinant adeno-associated virus (rAAV) vectors for gene therapy of inherited disorders have demonstrated considerable potential for molecular medicine. Recent identification of the viral receptor and coreceptors for AAV type 2 (AAV-2) has begun to explain why certain organs may demonstrate higher efficiencies of gene transfer with this vector. However, the mechanisms by which AAV-2 enters cells remain unknown. In the present report, we have examined whether the endocytic pathways of rAAV-2 are dependent on dynamin, a GTPase protein involved in clathrin-mediated internalization of r

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Sarukhan, Adelaida, Sabine Camugli, Bernard Gjata, Harald von Boehmer, Olivier Danos, and Karin Jooss. "Successful Interference with Cellular Immune Responses to Immunogenic Proteins Encoded by Recombinant Viral Vectors." Journal of Virology 75, no. 1 (2001): 269–77. http://dx.doi.org/10.1128/jvi.75.1.269-277.2001.

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ABSTRACT Vectors derived from the adeno-associated virus (AAV) have been successfully used for the long-term expression of therapeutic genes in animal models and patients. One of the major advantages of these vectors is the absence of deleterious immune responses following gene transfer. However, AAV vectors, when used in vaccination studies, can result in efficient humoral and cellular responses against the transgene product. It is therefore important to understand the factors which influence the establishment of these immune responses in order to design safe and efficient procedures for AAV-

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Galibert, Lionel, Aurélien Jacob, Adrien Savy, et al. "Monobac System–A Single Baculovirus for the Production of rAAV." Microorganisms 9, no. 9 (2021): 1799. http://dx.doi.org/10.3390/microorganisms9091799.

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Large-scale manufacturing of rAAV is a bottleneck for the development of genetic disease treatments. The baculovirus/Sf9 cell system underpins the first rAAV treatment approved by EMA and remains one of the most advanced platforms for rAAV manufacturing. Despite early successes, rAAV is still a complex biomaterial to produce. Efficient production of the recombinant viral vector requires that AAV replicase and capsid genes be co-located with the recombinant AAV genome. Here, we present the Monobac system, a singular, modified baculovirus genome that contains all of these functions. To assess th

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Bals, Robert, Weidong Xiao, Nianli Sang, Daniel J. Weiner, Rupalie L. Meegalla, and James M. Wilson. "Transduction of Well-Differentiated Airway Epithelium by Recombinant Adeno-Associated Virus Is Limited by Vector Entry." Journal of Virology 73, no. 7 (1999): 6085–88. http://dx.doi.org/10.1128/jvi.73.7.6085-6088.1999.

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ABSTRACT The limitations of adeno-associated virus (AAV)-mediated vectors for lung-directed gene transfer were investigated by using differentiated human respiratory epithelium in air-liquid interface cultures. Transduction efficiency was high in undifferentiated cells and was enhanced in well-differentiated cells after basolateral application of the vector or after apical application following disruption of tight junctions or pretreatment of the cultures with glycosidases. These results indicate that transduction of airway epithelia by AAV vectors is limited by entry and reinforce the importa

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31

Huang, Jiayin. "The Mechanism of Adeno-associated Virus (AAV) Gene Therapy." Highlights in Science, Engineering and Technology 123 (December 24, 2024): 84–89. https://doi.org/10.54097/ma9x1289.

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Adeno-associated Virus (AAV) vector has become an important tool for vivo gene therapy due to its high specificity, low immunogenicity and long-term expressibility. There are multiple novel pharmaceuticals utilizing recombinant AAV vectors to treat genetic defect disease or disease related to gene expression disorder that are undergoing clinical trials. In this review, the basic biological features of AAV viruses are introduced, including their structure, classification, and interactions with hosts. This paper aims to elucidate the commonalities and differences among several AAV viruses used i

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Duzenli, Ozgun Firat, and George Aslanidi. "Consecutive Affinity and Ion-Exchange Chromatography for AAV9 Vectors Purification." Biomedicines 13, no. 2 (2025): 361. https://doi.org/10.3390/biomedicines13020361.

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Background: Irrespective of the rapid development of AAV-based gene therapy, the production of clinical-grade vectors has a bottleneck resulting from product-related impurities such as empty and partially filled capsids, which lack a functional recombinant genome. Methods: In the current study, we applied the sequential affinity chromatography (AC)- and anion-exchange chromatography (AEX)-based method for purification of AAV9 vector harboring single-stranded genome encoding the fusion of firefly luciferase (fLuc)-yellow fluorescent protein (YFP) under chicken beta actin (CBA) promoter. We asse

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Wu, Ping, M. Ian Phillips, John Bui, and Ernest F. Terwilliger. "Adeno-Associated Virus Vector-Mediated Transgene Integration into Neurons and Other Nondividing Cell Targets." Journal of Virology 72, no. 7 (1998): 5919–26. http://dx.doi.org/10.1128/jvi.72.7.5919-5926.1998.

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ABSTRACT The site-specific integration of wild-type adeno-associated virus (wtAAV) into the human genome is a very attractive feature for the development of AAV-based gene therapy vectors. However, knowledge about integration of wtAAV, as well as currently configured recombinant AAV (rAAV) vectors, is limited. By using a modified Alu-PCR technique to amplify and sequence the vector-cellular junctions, we provide the first direct evidence both in vitro and in vivo of rAAV-mediated transgene integration in several types of nondividing cells, including neurons. This novel technique will be highly

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Stiefelhagen, Marius, Marlon R. Veldwijk, Anna Jauch, et al. "Generation and Application of a CML-Specific Recombinant Adeno-Associated Virus (rAAV) Vector." Blood 106, no. 11 (2005): 4417. http://dx.doi.org/10.1182/blood.v106.11.4417.4417.

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Abstract Introduction: Chronic myelogenous leukemia can be controlled but in most patients not be cured by tyrosine kinase inhibition. Direct targeting using gene therapy vectors combined with vaccination strategies may allow to eradicate residual leukemic progenitors. Adeno-associated virus (AAV) vectors are stable DNA vectors which were proven to be effective in the clinical gene therapy for e.g. coagulation disorders. The various AAV serotypes lack specificity for BCR-ABL+ leukemia cells. Recently developed AAV-library techniques allow a retargeting of vectors. We generated a set of rAAV ve

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Li, Chengwen, and R. Jude Samulski. "Serotype-specific replicating AAV helper constructs increase recombinant AAV type 2 vector production." Virology 335, no. 1 (2005): 10–21. http://dx.doi.org/10.1016/j.virol.2005.02.008.

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Davidoff, Andrew M., Catherine Y. C. Ng, Junfang Zhou, Yunyu Spence, and Amit C. Nathwani. "Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway." Blood 102, no. 2 (2003): 480–88. http://dx.doi.org/10.1182/blood-2002-09-2889.

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AbstractA systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice compared with female mice, irrespective of the proviral promoter or cDNA and mouse strain. Molecular analysis revealed that the rAAV genome was stably retained in male liver at levels that were 7-fold higher than those observed in females. Further, the sex difference in transduction was observed with AAV-2– and AAV-5–based vectors, which use

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Phillips, André W., Peisu Zhang, Mary Ellen Truckenmiller, et al. "Platelet-derived growth factor-producing cells immortalized from rat mesencephalon with SV40 large T antigen transduced by an AAV vector." Restorative Neurology and Neuroscience 21, no. 1-2 (2003): 1–10. https://doi.org/10.3233/rnn-2003-00220.

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Purpose: Adeno-associated virus (AAV) can infect a wide variety of mammalian cell types and is capable of infecting both dividing and non-dividing cell populations. Here we report the construction of a recombinant AAV vector which expresses the SV40 large T protein (AAV-T) and the use of this vector to immortalize primary cells from embryonic rat mesencephalon. Methods: The AAV-T vector was constructed by introducing the BamH1 fragment of the pCMV/SVE/Neo plasmid containing T antigen and SV40 regulatory elements into the JM48 plasmid containing the inverted terminal repeats of AAV. Neuronal cu

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Guilbaud, Mickaël, Gilliane Chadeuf, Fabio Avolio, et al. "Relative Influence of the Adeno-Associated Virus (AAV) Type 2 p5 Element for Recombinant AAV Vector Site-Specific Integration." Journal of Virology 82, no. 5 (2007): 2590–93. http://dx.doi.org/10.1128/jvi.01956-07.

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ABSTRACT The p5 promoter region of the adeno-associated virus type 2 (AAV-2) rep gene has been described as essential for Rep-mediated site-specific integration (RMSSI) of plasmid sequences in human chromosome 19. We report here that insertion of a full-length or minimal p5 element between the viral inverted terminal repeats does not significantly increase RMSSI of a recombinant AAV (rAAV) vector after infection of growth-arrested or proliferating human cells. This result suggests that the p5 element may not improve RMSSI of rAAV vectors in vivo.

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Wendtner, Clemens-Martin, David M. Kofler, Hans D. Theiss, et al. "Efficient gene transfer of CD40 ligand into primary B-CLL cells using recombinant adeno-associated virus (rAAV) vectors." Blood 100, no. 5 (2002): 1655–61. http://dx.doi.org/10.1182/blood.v100.5.1655.h81702001655_1655_1661.

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B cells of chronic lymphocytic leukemia (B-CLL) are resistant to transduction with most currently available vector systems. Using an optimized adenovirus-free packaging system, recombinant adeno-associated virus (rAAV) vectors coding for the enhanced green fluorescent protein (AAV/EGFP) and CD40 ligand (AAV/CD40L) were packaged and highly purified resulting in genomic titers up to 3 × 1011/mL. Cells obtained from 24 patients with B-CLL were infected with AAV/EGFP or AAV/CD40L at a multiplicity of infection (MOI) of 100 resulting in transgene expression in up to 97% of cells as detected by flow

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Wang, Fei, Jiawen Sun, Wenyan Guo, and Yang Wu. "Application of the Insect Cell-Baculovirus Expression Vector System in Adeno-Associated Viral Production." Applied Sciences 14, no. 23 (2024): 10948. http://dx.doi.org/10.3390/app142310948.

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Insect Cell-Baculovirus Expression Vector System (IC-BEVS) is an efficient protein expression platform, which is famous for its high-level expression of complex protein in insect cells. The system is based on baculoviruses such as Autographa californica multiple nucleopolyhedrovirus (AcMNPV), and the expression efficiency of the target proteins has been significantly improved by optimizing the viral vectors and cell lines. In recent years, IC-BEVS have shown great potential for Adeno-Associated Virus (AAV) production, particularly excelling in AAV structural protein expression and recombinant

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Akil, Omar, Frank Dyka, Charlotte Calvet, et al. "Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model." Proceedings of the National Academy of Sciences 116, no. 10 (2019): 4496–501. http://dx.doi.org/10.1073/pnas.1817537116.

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Autosomal recessive genetic forms (DFNB) account for most cases of profound congenital deafness. Adeno-associated virus (AAV)-based gene therapy is a promising therapeutic option, but is limited by a potentially short therapeutic window and the constrained packaging capacity of the vector. We focus here on the otoferlin gene underlying DFNB9, one of the most frequent genetic forms of congenital deafness. We adopted a dual AAV approach using two different recombinant vectors, one containing the 5′ and the other the 3′ portions of otoferlin cDNA, which exceed the packaging capacity of the AAV wh

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Xiao, Xiao, Juan Li, and Richard Jude Samulski. "Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus." Journal of Virology 72, no. 3 (1998): 2224–32. http://dx.doi.org/10.1128/jvi.72.3.2224-2232.1998.

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ABSTRACT Recently, efficient and long-term in vivo gene transfer by recombinant adeno-associated virus type 2 (rAAV) vectors has been demonstrated in a variety of tissues. Further improvement in vector titer and purity will expedite this in vivo exploration and provide preclinical information required for use in human gene therapy. In an effort to obtain higher titers, we constructed a novel AAV helper plasmid which utilizes translational control of AAV Rep genes (J. Li et al., J. Virol. 71:5236–5243, 1997). To address the issue of purity, in this study we report the first rAAV production meth

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Dong, Biao, Hiroyuki Nakai, and Weidong Xiao. "Characterization of Genome Integrity for Oversized Recombinant AAV Vector." Molecular Therapy 18, no. 1 (2010): 87–92. http://dx.doi.org/10.1038/mt.2009.258.

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Deodato, B., N. Arsic, L. Zentilin, et al. "Recombinant AAV vector encoding human VEGF165 enhances wound healing." Gene Therapy 9, no. 12 (2002): 777–85. http://dx.doi.org/10.1038/sj.gt.3301697.

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Cao, L., M. During, and W. Xiao. "Replication competent helper functions for recombinant AAV vector generation." Gene Therapy 9, no. 18 (2002): 1199–206. http://dx.doi.org/10.1038/sj.gt.3301710.

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Grimm, Dirk, Joyce S. Lee, Lora Wang, et al. "In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses." Journal of Virology 82, no. 12 (2008): 5887–911. http://dx.doi.org/10.1128/jvi.00254-08.

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ABSTRACT Adeno-associated virus (AAV) serotypes differ broadly in transduction efficacies and tissue tropisms and thus hold enormous potential as vectors for human gene therapy. In reality, however, their use in patients is restricted by prevalent anti-AAV immunity or by their inadequate performance in specific targets, exemplified by the AAV type 2 (AAV-2) prototype in the liver. Here, we attempted to merge desirable qualities of multiple natural AAV isolates by an adapted DNA family shuffling technology to create a complex library of hybrid capsids from eight different wild-type viruses. Sel

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Ogasawara, Yoji, Hiroaki Mizukami, Masashi Urabe, et al. "Highly regulated expression of adeno-associated virus large Rep proteins in stable 293 cell lines using the Cre/loxP switching system." Journal of General Virology 80, no. 9 (1999): 2477–80. http://dx.doi.org/10.1099/0022-1317-80-9-2477.

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Since the Rep proteins of adeno-associated virus (AAV) are harmful to cells, it is difficult to obtain stable cell lines that express them constitutively. In this study, stable 293 cell lines were obtained in which large Rep expression was inducible by using the Cre/loxP switching system. To determine the function of the induced Rep proteins, the packaging capacity was examined after supplementation with a plasmid expressing small Rep and Cap proteins. A significant amount of recombinant AAV (5·5×108 vector particles per 10 cm dish) was produced by transfection with a vector plasmid and infect

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Hüser, Daniela, Stefan Weger, and Regine Heilbronn. "Packaging of Human Chromosome 19-Specific Adeno-Associated Virus (AAV) Integration Sites in AAV Virions during AAV Wild-Type and Recombinant AAV Vector Production." Journal of Virology 77, no. 8 (2003): 4881–87. http://dx.doi.org/10.1128/jvi.77.8.4881-4887.2003.

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ABSTRACT Adeno-associated virus type 2 (AAV-2) establishes latency by site-specific integration into a unique locus on human chromosome 19, called AAVS1. During the development of a sensitive real-time PCR assay for site-specific integration, AAV-AAVS1 junctions were reproducibly detected in highly purified AAV wild-type and recombinant AAV vector stocks. A series of controls documented that the junctions were packaged in AAV capsids and were newly generated during a single round of AAV production. Cloned junctions displayed variable AAV sequences fused to AAVS1. These data suggest that packag

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Zhang, Yi, Narendra Chirmule, Guang-ping Gao, and James Wilson. "CD40 Ligand-Dependent Activation of Cytotoxic T Lymphocytes by Adeno-Associated Virus Vectors In Vivo: Role of Immature Dendritic Cells." Journal of Virology 74, no. 17 (2000): 8003–10. http://dx.doi.org/10.1128/jvi.74.17.8003-8010.2000.

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ABSTRACT Recombinant adeno-associated virus type 2 (rAAV) is being explored as a vector for gene therapy because of its broad host range, good safety profile, and persistent transgene expression in vivo. However, accumulating evidence indicates that administration of AAV vector may initiate a detectable cellular and humoral immune response to its transduced neo-antigen in vivo. To elucidate the cellular basis of the AAV-mediated immune response, C57BL/6 mouse bone marrow-derived immature and mature dendritic cells (DCs) were infected with AAV encoding β-galactosidase (AAV-lacZ) and adoptively

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George, Lindsey A. "Hemophilia gene therapy: ushering in a new treatment paradigm?" Hematology 2021, no. 1 (2021): 226–33. http://dx.doi.org/10.1182/hematology.2021000254.

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Abstract After 3 decades of clinical trials, repeated proof-of-concept success has now been demonstrated in hemophilia A and B gene therapy. Current clinical hemophilia gene therapy efforts are largely focused on the use of systemically administered recombinant adeno-associated viral (rAAV) vectors for F8 or F9 gene addition. With multiple ongoing trials, including licensing studies in hemophilia A and B, many are cautiously optimistic that the first AAV vectors will obtain regulatory approval within approximately 1 year. While supported optimism suggests that the goal of gene therapy to alter

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