Self complementary aav patent

Author: jmhd

August undefined, 2024

WebAbstract Using a mouse model we show that self-complementary (sc) adeno-associated virus (AAV) vectors pseudotyped with capsids of serotypes 2, 7 or 8 induce more potent transgene product-specific CD8 (+) T cell and antibody responses compared to corresponding single-stranded (ss)AAV vectors. WebSelf-complementary AAV vectors were titered by the ddPCR method, using the eGFP assay in the presence or absence of DNase I, and in all cases the titers were similar (Supplementary Table S1;...

RNAi-mediated rheostat for dynamic control of AAV-delivered …

WebNov 23, 2024 · Sensorineural hearing loss is one of the most common disabilities worldwide. Such prevalence necessitates effective tools for studying the molecular workings of cochlear cells. One prominent and effective vector for expressing genes of interest in research models is adeno-associated virus (AAV). However, AAV efficacy in transducing … WebThe AAV vector may be of any serotype (e.g., type 1, type 2, type 3, type 4, type 5, type 6, type 7, type 8, type 9, type 10). In another embodiment, a self-complementary vector carrying the smCBA promoter is administered to the eye. Another aspect of the invention concerns host cells carrying a vector of the invention. emmylou harris songs list

Engineering adeno-associated virus vectors for gene

WebL'apparition de différences dans le texte et l'image des Revendications et de l'Abrégé dépend du moment auquel le document est publié. Les textes des Revendications et de l'Abrégé sont affichés : Webthe normal AAV packaging capacity (^4,700 nucleotides). In this case, the vector product is a mixture, containing virions with self-complementary genomes and virions that have packaged either one or two, monomeric ssDNA molecules. The proportion of dimeric to monomeric genomes varies widely between preps, from ^5 to >50% dimer. Weba complementary strand in order to achieve gene expression. This is the critical step that can be effectively bypassed through the use of self-complementary AAV (scAAV) vectors. … drain surround wickes

Adeno-associated virus terminal repeat (TR) mutant generates self …

Addgene: pscAAV-CAG-GFP

WebAug 30, 2016 · US Patent for AAV-EPO for treating companion animals Patent (Patent # 11,117,942) AAV-EPO for treating companion animals Aug 30, 2016 - The Trustees of the University of Pennsylvania Compositions and methods are provided for treating companion animals are provided. WebOct 1, 2008 · The increases in efficiency gained with self-complementary AAV (scAAV) vectors have ranged from modest to stunning, depending on the tissue, cell type, and … drain surveyingWebNov 20, 2014 · In a variety of animal models, 4-6 vectors that are based on adeno-associated virus (AAV), a nonpathogenic parvovirus, have shown the greatest promise for gene therapy in patients with hemophilia... drain surround concrete

"WebFeb 10, 2024 · Using synthetic poly (A) can increase the nuclear export, translation and stability of mRNA (by enhancing polyadenylation), and using reversed poly (A) can avoid the transcription of ITR; both... " - Self complementary aav patent

Self complementary aav patent

Self-complementary AAV Vectors; Advances and Applications

Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy. Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases. This lab-made progeny of rAAV is termed "self-complementary" because the coding region has been designed to form an intra-molecular double-stranded DNA template. A rate-limiting step for the standard AAV … WebApr 8, 2024 · In line with our in vitro findings, expression of the self-silencing AAV containing both an intronic miR-33 TTR shRNA and its complementary binding site (TTR shRNA/TTR-ts) led to a sustained ...

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WebView Patent Images: Download PDF 20240098714 . Export Citation: Click for automatic bibliography generation . Assignee: New York R&D Center for Translational Medicine and Therapeutics, Inc. (New Rochelle, NY, US) International Classes: C12N15/113; A61P19/04. Primary Examiner: ...

WebIn another embodiment, the vector carrying the smCBA promoter is a self- complementary adeno-associated virus (AAV). The AAV vector may be of any serotype (e.g., type 1, type 2, type 3, type 4, type 5, type 6, type 7, type 8, type 9, type 10). In another embodiment, a self-complementary vector carrying the smCBA promoter is administered to the eye. WebNov 23, 2024 · Reports in other systems suggest multiple approaches can be used to enhance AAV transduction including self-complementary vector design and …

WebSelf-complementary adeno-associated virus (scAAV) contains a dimeric inverted repeat genome able to fold into double stranded DNA (dsDNA). scAAV particles possess several … WebThe enhanced vector constructs are highly flexible, user-friendly, and can be easily modified (via routine DNA cloning) and utilized (via standard AAV vector technology) by anyone …

WebFeb 9, 2024 · In order to overcome this problem, self-complementary (sc) AAV vectors have been introduced as these vectors contain a dimeric inverted repeat genome that allows folding into dsDNA ... and is currently patent-free . Notably, each disease group appears to have a capsid preference, likely due to the different tissue tropism each serotype exhibits

WebJan 31, 2006 · US Patent Application for Self-complementary parvoviral vectors, and methods for making and using the same Patent Application (Application #20070253936 issued November 1, 2007) - Justia Patents Search Self-complementary parvoviral vectors, and methods for making and using the same Jan 31, 2007 drains winsfordWebRecombinant AAV vectors have predominantly episomal gene expression. AAV has long term expression in terminally differentiated cells. It can infect a wide range of cells, including non-dividing cells. Advantage of the Self-Complementary AAV Plasmid: The efficiency of AAV vectors is “hindered by the need to convert the single-stranded DNA drain surroundWebAbstract. purpose. Self-complementary AAV (scAAV) vectors have been developed to circumvent rate-limiting second-strand synthesis in single-stranded AAV vector genomes … dr ainsworth newcastleWebNov 23, 2024 · Reports in other systems suggest multiple approaches can be used to enhance AAV transduction including self-complementary vector design and … drain survey yorkWebDec 6, 2024 · Abstract. Gene therapy offers the potential for a cure for patients with hemophilia of establishing continuous endogenous print out factor EIGHT or factor IX drain tackle 2 blitzballWebAAV variants: Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. McCarty DM, Monahan … dr. ainsworthWebIn particular, CRISPR-Cas9 gene editing components packaged by self-complementary AAV (scAAV) demonstrate robust viral transduction and efficient gene editing, enabling restoration of dystrophin expression throughout skeletal and cardiac muscle in animal models of DMD. Here, we describe protocols for cloning CRISPR single guide RNAs … drains wigan