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• W2365630593 abstract "Small promoters that recapitulate endogenous gene expression patterns are important for basic, preclinical, and now clinical research. Recently, there has been a promising revival of gene therapy for diseases with unmet therapeutic needs. To date, most gene therapies have used viral-based ubiquitous promoters–however, promoters that restrict expression to target cells will minimize off-target side effects, broaden the palette of deliverable therapeutics, and thereby improve safety and efficacy. Here, we take steps towards filling the need for such promoters by developing a high-throughput pipeline that goes from genome-based bioinformatic design to rapid testing in vivo. For much of this work, therapeutically interesting Pleiades MiniPromoters (MiniPs; ~4 kb human DNA regulatory elements), previously tested in knock-in mice, were “cut down” to ~2.5 kb and tested in recombinant adeno-associated virus (rAAV), the virus of choice for gene therapy of the central nervous system. To evaluate our methods, we generated 29 experimental rAAV2/9 viruses carrying 19 different MiniPs, which were injected intravenously into neonatal mice to allow broad unbiased distribution, and characterized in neural tissues by X-gal immunohistochemistry for icre, or immunofluorescent detection of GFP. The data showed that 16 of the 19 (84 %) MiniPs recapitulated the expression pattern of their design source. This included expression of: Ple67 in brain raphe nuclei; Ple155 in Purkinje cells of the cerebellum, and retinal bipolar ON cells; Ple261 in endothelial cells of brain blood vessels; and Ple264 in retinal Müller glia. Overall, the methodology and MiniPs presented here represent important advances for basic and preclinical research, and may enable a paradigm shift in gene therapy." @default.
• W2365630593 created "2016-06-24" @default.
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• W2365630593 date "2016-05-10" @default.
• W2365630593 modified "2023-09-26" @default.
• W2365630593 title "rAAV-compatible MiniPromoters for restricted expression in the brain and eye" @default.
• W2365630593 cites W1497068177 @default.
• W2365630593 cites W1544691147 @default.
• W2365630593 cites W1569867067 @default.
• W2365630593 cites W1601243018 @default.
• W2365630593 cites W1769569140 @default.
• W2365630593 cites W1850657797 @default.
• W2365630593 cites W1902159756 @default.
• W2365630593 cites W1903485451 @default.
• W2365630593 cites W1934209312 @default.
• W2365630593 cites W1964733653 @default.
• W2365630593 cites W1967308064 @default.
• W2365630593 cites W1970839656 @default.
• W2365630593 cites W1977035092 @default.
• W2365630593 cites W1981877907 @default.
• W2365630593 cites W1989073393 @default.
• W2365630593 cites W1992203015 @default.
• W2365630593 cites W1993489714 @default.
• W2365630593 cites W1995649219 @default.
• W2365630593 cites W1995848848 @default.
• W2365630593 cites W2003572485 @default.
• W2365630593 cites W2009935171 @default.
• W2365630593 cites W2009963071 @default.
• W2365630593 cites W2011457085 @default.
• W2365630593 cites W2012254260 @default.
• W2365630593 cites W2012894204 @default.
• W2365630593 cites W2013878893 @default.
• W2365630593 cites W2015741711 @default.
• W2365630593 cites W2017990041 @default.
• W2365630593 cites W2024826635 @default.
• W2365630593 cites W2026277721 @default.
• W2365630593 cites W2031344272 @default.
• W2365630593 cites W2033201031 @default.
• W2365630593 cites W2035747410 @default.
• W2365630593 cites W2035792918 @default.
• W2365630593 cites W2037005126 @default.
• W2365630593 cites W2042183771 @default.
• W2365630593 cites W2049439326 @default.
• W2365630593 cites W2051442157 @default.
• W2365630593 cites W2052280552 @default.
• W2365630593 cites W2057568687 @default.
• W2365630593 cites W2068666140 @default.
• W2365630593 cites W2073566204 @default.
• W2365630593 cites W2077683647 @default.
• W2365630593 cites W2083073732 @default.
• W2365630593 cites W2086705852 @default.
• W2365630593 cites W2088338126 @default.
• W2365630593 cites W2111105486 @default.
• W2365630593 cites W2111164126 @default.
• W2365630593 cites W2118596665 @default.
• W2365630593 cites W2123491442 @default.
• W2365630593 cites W2125795987 @default.
• W2365630593 cites W2126115955 @default.
• W2365630593 cites W2126939295 @default.
• W2365630593 cites W2138363022 @default.
• W2365630593 cites W2139148195 @default.
• W2365630593 cites W2140664826 @default.
• W2365630593 cites W2142717029 @default.
• W2365630593 cites W2146734921 @default.
• W2365630593 cites W2152069082 @default.
• W2365630593 cites W2152853777 @default.
• W2365630593 cites W2159358696 @default.
• W2365630593 cites W2160379721 @default.
• W2365630593 cites W2160426928 @default.
• W2365630593 cites W2165064122 @default.
• W2365630593 cites W2166739815 @default.
• W2365630593 cites W2172096124 @default.
• W2365630593 cites W2247766769 @default.
• W2365630593 cites W2259938310 @default.
• W2365630593 cites W2465346652 @default.
• W2365630593 cites W4234522512 @default.

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