CONTROLLED EXPRESSION OF TRANSGENES USING CLOSE-ENDED DNA (CEDNA) VECTORS

Provided herein are methods and constructs comprising close-ended DNA (ceDNA vectors) for maintaining or sustaining a level of transgene expression at a predetermined level or range for a predefined time, or increasing the level of transgene expression in a cell or a subject, where the transgene exp...

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Hauptverfasser: CHIOCCO MATT, SAMAYOA PHILLIP, KERR DOUGLAS A, STANTON MATTHEW G, KOTIN ROBERT M, ANGELINO MARK D
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creator CHIOCCO MATT
SAMAYOA PHILLIP
KERR DOUGLAS A
STANTON MATTHEW G
KOTIN ROBERT M
ANGELINO MARK D
description Provided herein are methods and constructs comprising close-ended DNA (ceDNA vectors) for maintaining or sustaining a level of transgene expression at a predetermined level or range for a predefined time, or increasing the level of transgene expression in a cell or a subject, where the transgene expression level can be modulated (e.g., increased) with one or more subsequent administrations (e.g.,a re-dose or a booster administration) after an initial priming administration. Provided are methods for personalizing gene therapy throughout an individuals' lifespan to express a transgene at a level that meets an individual's needs, by modulating expression levels of a transgene expressed by ceDNA vector incrementally, or in a step-by-step manner, with one or more administrations after an initial priming administration (e.g., at time 0), thereby enabling titration of the level of expression of the transgene to a desired predetermined expression level or to a desired expression level range. 本文提供了包含闭合端DNA的方法和构建体(ceDN
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language chi ; eng
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
title CONTROLLED EXPRESSION OF TRANSGENES USING CLOSE-ENDED DNA (CEDNA) VECTORS
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