Bibliografías temáticas / AAV recombinant vector

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Literatura académica sobre el tema "AAV recombinant vector"

Autor: Grafiati
Publicado: 25 de enero de 2025
Última modificación: 31 de julio de 2025

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Artículos de revistas sobre el tema "AAV recombinant vector"

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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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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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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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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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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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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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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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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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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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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Tesis sobre el tema "AAV recombinant vector"

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Carty, Nikisha Christine. "Recombinant AAV Gene Therapy and Delivery." Scholar Commons, 2009. https://scholarcommons.usf.edu/etd/1890.

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Alzheimer's disease (AD), first characterized in the early 20th century, is a common form of dementia which can occur as a result of genetic mutations in the genes encoding presenilin 1, presenilin 2, or amyloid precursor protein (APP). These genetic alterations can accelerate the pathological characteristics of AD, including the formation of extracellular neuritic plaques composed of amyloid beta peptides and the formation of intracellular neurofibrillary tangles consisting of hyperphosphorylated tau protein. Ultimately, AD results in gross neuron loss in the brain which is evidenced clinical
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2

Xu, Dan. "Cellular Immunity in Recombinant Adeno-Associated Virus Vector Mediated Gene Therapy." The Ohio State University, 2011. http://rave.ohiolink.edu/etdc/view?acc_num=osu1313504203.

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Jeanpierre, Lindsay. "Etude des réponses T CD8+ après transfert de gène par rAAV ciblant le muscle." Electronic Thesis or Diss., université Paris-Saclay, 2024. https://www.biblio.univ-evry.fr/theses/2024/interne/2024UPASL128.pdf.

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La thérapie génique par vecteurs dérivés des virus adéno-associés (rAAV), s'est imposée ces dernières décennies, comme une stratégie de traitement innovante, pour le traitement des maladies monogéniques rares. Elle est basée sur l'utilisation d'un vecteur d'origine virale, pour délivrer une séquence d'ADN fonctionnelle, dans les cellules présentant la mutation à l'origine de la maladie.Les avancées dans ce domaine ont conduit à la mise sur le marché de plusieurs produits, dont le ZolgenSMA®, utilisé pour le traitement de l'amyotrophie spinale, qui a permis de soigner plus de 4000 patients dans
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Ghenassia, Alexandre. "Induction de réponses mémoires lymphocytaires T CD8 et protection vaccinale après transfert de gènes par le vecteur AAV recombinant." Thesis, Sorbonne Paris Cité, 2015. http://www.theses.fr/2015PA05T032/document.

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La mémoire immunologique est le mécanisme biologique fondamental à la base du développement de la vaccination. La compréhension de ce mécanisme ainsi que de ses interactions avec les différents acteurs du système immunitaire a permis l’élaboration de vaccins qui sont aujourd’hui les garants d’une protection accrue face à l’émergence de maladies infectieuses potentiellement mortelles. La voie d’injection et le mode de transfert de ces vaccins sont des paramètres majeurs à prendre en considération car ils définissent une modulation des réponses immunitaires et de leurs spécificités d’action. De
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Ploquin, Aurélie. "Les vecteurs AAV recombinants : un nouvel outil de vaccination contre les Hénipavirus." Phd thesis, Ecole normale supérieure de lyon - ENS LYON, 2012. http://tel.archives-ouvertes.fr/tel-00756311.

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Les virus Hendra (HeV) et Nipah (NiV) sont des virus émergents appartenant à la famille des Paramyxovirus et au genre des Hénipavirus. Chaque année, ils sont responsables de nombreuses épidémies touchant plusieurs espèces animales dont les hommes, avec une forte morbidité et mortalité. À ce jour, aucun vaccin ni traitement ne sont commercialisés. Ce projet porte sur le développement d'un vaccin génétique pour lutter contre une infection par les Hénipavirus. La stratégie suivie, repose sur l'injection in vivo de vecteurs recombinants dérivés du virus Adéno-Associé (AAVr) codant pour la glycopro
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Özgür, Günes Yasemin. "Preclinical gene therapy using recombinant AAV vectors in mouse models of two human diseases." Thesis, université Paris-Saclay, 2022. http://www.theses.fr/2022UPASL092.

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Nous avons obtenu une preuve de concept pour la thérapie génique de 2 maladies génétiques perte-de-fonction.L'acrodysostose est une maladie osseuse et rénale causée par des mutations de la sous-unité régulatrice de la protéine kinase A (PRKAR1A). Nous avons testé les effets d'un rAAV9-CAG-humanPRKR1A dans un modèle murin KI. hPRKAR1A a été exprimée dans les chondrocytes de la plaque de croissance et les cellules tubulaires rénales. L'architecture des chondrocytes et la longueur du squelette ont été améliorées.L'AMN est une axonopathie tardive de la moelle épinière causée par des mutations du g
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Best, Victoria Maria. "Ongoing cellular responses to transgene products encoded by recombinant adeno-associated virus (rAAV) vectors." The Ohio State University, 2009. http://rave.ohiolink.edu/etdc/view?acc_num=osu1262213552.

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Niemir, Natalia. "Gene transfer in the Sandhoff murine model using a specific recombinant AAV9 vector." Thesis, Paris 5, 2013. http://www.theses.fr/2013PA05S024/document.

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Koo, Taeyoung. "Studies on gene transfer in skeletal muscle cells and tissues using recombinant adeno-associated virus (AAV) vectors." Thesis, Royal Holloway, University of London, 2010. http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.529039.

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Chtarto, Abdelwahed. "Contribution au développement de nouveaux vecteurs inductibles par la tétracycline et basés sur le parvovirus adéno-associé (AAV)." Doctoral thesis, Universite Libre de Bruxelles, 2005. http://hdl.handle.net/2013/ULB-DIPOT:oai:dipot.ulb.ac.be:2013/210983.

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Le parvovirus adéno-associé (AAV) possède un génome à ADN linéaire simple brin de 4,7kb encadré par deux séquences palindromiques inversées et identiques de 145 nucléotides appelées ITRs, requises en cis pour la réplication et l’encapsidation de l’ADN viral. Dans un AAV recombinant (rAAV), la totalité de la partie codante du génome viral est remplacée par une cassette d’expression et seuls les ITRs sont conservés.<p>\<br>Doctorat en sciences biomédicales<br>info:eu-repo/semantics/nonPublished
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Capítulos de libros sobre el tema "AAV recombinant vector"

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Hallek, M., C. M. Wendtner, R. Kotin, D. Michl, and E. L. Winnacker. "Recombinant Adeno-Associated Virus (r AAV) Vectors." In Gene Therapy. Birkhäuser Basel, 1999. http://dx.doi.org/10.1007/978-3-0348-7011-5_6.

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Murlidharan, Giridhar, R. Jude Samulski, and Aravind Asokan. "Gene Therapy of CNS Disorders Using Recombinant AAV Vectors." In Translational Neuroscience. Springer US, 2016. http://dx.doi.org/10.1007/978-1-4899-7654-3_2.

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Lebkowski, J. S., T. B. Okarma, and R. Philip. "The Challenges of Recombinant Adeno-associated Virus Manufacturing: Alternative Use of Adeno-associated Virus Plasmid/Liposome Complexes for Gene Therapy Applications." In Adeno-Associated Virus (AAV) Vectors in Gene Therapy. Springer Berlin Heidelberg, 1996. http://dx.doi.org/10.1007/978-3-642-80207-2_4.

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Gupta, Megha. "Parvovirus Vectors: The Future of Gene Therapy." In Veterinary Medicine and Science. IntechOpen, 2022. http://dx.doi.org/10.5772/intechopen.105085.

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The unique diversity of parvoviral vectors with innate antioncogenic properties, autonomous replication, ease of recombinant vector production and stable transgene expression in target cells makes them an attractive choice as viral vectors for gene therapy protocols. Amongst various parvoviruses that have been identified so far, recombinant vectors originating from adeno-associated virus, minute virus of mice (MVM), LuIII and parvovirus H1 have shown promising results in many preclinical models of human diseases including cancer. The adeno-associated virus (AAV), a non-pathogenic human parvovi
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Yan, Ziying, Teresa C. Ritchie, Dongsheng Duan, and John F. Engelhardt. "[20] Recombinant AAV-mediated gene delivery using dual vector heterodimerization." In Methods in Enzymology. Elsevier, 2002. http://dx.doi.org/10.1016/s0076-6879(02)46065-x.

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R. Flotte, Terence. "Recombinant AAV vectors for gene transfer to the lung: a compartmental approach." In Adeno-Associated Viral Vectors for Gene Therapy. Elsevier, 2005. http://dx.doi.org/10.1016/s0075-7535(05)31004-7.

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Actas de conferencias sobre el tema "AAV recombinant vector"

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Chulay, J., M. Brantly, L. Wang, et al. "Clinical Evaluation of a Recombinant Adeno-Associated Virus (rAAV) Alpha-1 Antitrypsin (AAT) Gene Therapy Vector." In American Thoracic Society 2009 International Conference, May 15-20, 2009 • San Diego, California. American Thoracic Society, 2009. http://dx.doi.org/10.1164/ajrccm-conference.2009.179.1_meetingabstracts.a2908.

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Flotte, Terence, Bruce Trapnell, Margaret Humphries, et al. "Phase 2 Clinical Trial Of A Recombinant Adeno-Associated Virus (RAAV) Alpha-1 Antitrypsin (AAT) Gene Therapy Vector." In American Thoracic Society 2011 International Conference, May 13-18, 2011 • Denver Colorado. American Thoracic Society, 2011. http://dx.doi.org/10.1164/ajrccm-conference.2011.183.1_meetingabstracts.a2428.

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