Matches in SemOpenAlex for { <https://semopenalex.org/work/W4205120560> ?p ?o ?g. }
- W4205120560 endingPage "857" @default.
- W4205120560 startingPage "842" @default.
- W4205120560 abstract "In amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, both cell-autonomous and noncell-autonomous mechanisms lead to the selective degeneration of motoneurons (MN). Here, we evaluate the therapeutic potential of gene therapy targeting mutated SOD1 in mature astrocytes using mice expressing the mutated SOD1G93A protein. An AAV-gfaABC1 D vector encoding an artificial microRNA is used to deliver RNA interference against mutated SOD1 selectively in astrocytes. The treatment leads to the progressive rescue of neuromuscular junction occupancy, to the recovery of the compound muscle action potential in the gastrocnemius muscle, and significantly improves neuromuscular function. In the spinal cord, gene therapy targeting astrocytes protects a small pool of the most vulnerable fast-fatigable MN until disease end stage. In the gastrocnemius muscle of the treated SOD1G93A mice, the fast-twitch type IIB muscle fibers are preserved from atrophy. Axon collateral sprouting is observed together with muscle fiber type grouping indicative of denervation/reinnervation events. The transcriptome profiling of spinal cord MN shows changes in the expression levels of factors regulating the dynamics of microtubules. Gene therapy delivering RNA interference against mutated SOD1 in astrocytes protects fast-fatigable motor units and thereby improves neuromuscular function in ALS mice." @default.
- W4205120560 created "2022-01-26" @default.
- W4205120560 creator A5000792302 @default.
- W4205120560 creator A5001356149 @default.
- W4205120560 creator A5004386868 @default.
- W4205120560 creator A5043051104 @default.
- W4205120560 creator A5049921490 @default.
- W4205120560 creator A5060808313 @default.
- W4205120560 creator A5074420172 @default.
- W4205120560 creator A5082525881 @default.
- W4205120560 date "2022-01-03" @default.
- W4205120560 modified "2023-10-09" @default.
- W4205120560 title "Astrocyte‐targeting <scp>RNA</scp> interference against mutated superoxide dismutase 1 induces motoneuron plasticity and protects fast‐fatigable motor units in a mouse model of amyotrophic lateral sclerosis" @default.
- W4205120560 cites W1512899315 @default.
- W4205120560 cites W1915809219 @default.
- W4205120560 cites W1964382920 @default.
- W4205120560 cites W1970574518 @default.
- W4205120560 cites W1971793795 @default.
- W4205120560 cites W1977978745 @default.
- W4205120560 cites W1979837223 @default.
- W4205120560 cites W1983232447 @default.
- W4205120560 cites W1984861969 @default.
- W4205120560 cites W1990078383 @default.
- W4205120560 cites W1991323513 @default.
- W4205120560 cites W1996024251 @default.
- W4205120560 cites W1999574084 @default.
- W4205120560 cites W2008092479 @default.
- W4205120560 cites W2015153408 @default.
- W4205120560 cites W2024497434 @default.
- W4205120560 cites W2026147918 @default.
- W4205120560 cites W2027047473 @default.
- W4205120560 cites W2027178374 @default.
- W4205120560 cites W2033158384 @default.
- W4205120560 cites W2033167804 @default.
- W4205120560 cites W2034850763 @default.
- W4205120560 cites W2036483462 @default.
- W4205120560 cites W2036897871 @default.
- W4205120560 cites W2038963685 @default.
- W4205120560 cites W2043576437 @default.
- W4205120560 cites W2045492362 @default.
- W4205120560 cites W2046615897 @default.
- W4205120560 cites W2051373450 @default.
- W4205120560 cites W2059355164 @default.
- W4205120560 cites W2059473556 @default.
- W4205120560 cites W2060230550 @default.
- W4205120560 cites W2062808481 @default.
- W4205120560 cites W2064396921 @default.
- W4205120560 cites W2066557327 @default.
- W4205120560 cites W2069498125 @default.
- W4205120560 cites W2070720564 @default.
- W4205120560 cites W2080209477 @default.
- W4205120560 cites W2090977212 @default.
- W4205120560 cites W2098622411 @default.
- W4205120560 cites W2107660300 @default.
- W4205120560 cites W2107768418 @default.
- W4205120560 cites W2109192152 @default.
- W4205120560 cites W2111027979 @default.
- W4205120560 cites W2112445405 @default.
- W4205120560 cites W2112952507 @default.
- W4205120560 cites W2121396070 @default.
- W4205120560 cites W2124976615 @default.
- W4205120560 cites W2127488964 @default.
- W4205120560 cites W2132315578 @default.
- W4205120560 cites W2134526812 @default.
- W4205120560 cites W2137586531 @default.
- W4205120560 cites W2141759311 @default.
- W4205120560 cites W2144977714 @default.
- W4205120560 cites W2149497936 @default.
- W4205120560 cites W2160249662 @default.
- W4205120560 cites W2161239543 @default.
- W4205120560 cites W2166043619 @default.
- W4205120560 cites W2167912995 @default.
- W4205120560 cites W2169456326 @default.
- W4205120560 cites W2170485551 @default.
- W4205120560 cites W2179438025 @default.
- W4205120560 cites W2180571620 @default.
- W4205120560 cites W2290358349 @default.
- W4205120560 cites W2297831727 @default.
- W4205120560 cites W2303102322 @default.
- W4205120560 cites W2334833725 @default.
- W4205120560 cites W2413296873 @default.
- W4205120560 cites W2497005327 @default.
- W4205120560 cites W2520485831 @default.
- W4205120560 cites W2556818673 @default.
- W4205120560 cites W2582442897 @default.
- W4205120560 cites W2604498541 @default.
- W4205120560 cites W2735021289 @default.
- W4205120560 cites W2753394810 @default.
- W4205120560 cites W2795357920 @default.
- W4205120560 cites W2797148904 @default.
- W4205120560 cites W2810622900 @default.
- W4205120560 cites W2884390718 @default.
- W4205120560 cites W2907108807 @default.
- W4205120560 cites W2910480975 @default.
- W4205120560 cites W2929622128 @default.
- W4205120560 cites W2933621474 @default.
- W4205120560 cites W2966772209 @default.
- W4205120560 cites W3044057346 @default.