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- W2944990939 endingPage "262" @default.
- W2944990939 startingPage "248" @default.
- W2944990939 abstract "Nucleotide repeat disorders encompass more than 30 diseases, most of which show dominant inheritance, such as Huntington’s disease, spinocerebellar ataxias, and myotonic dystrophies. Yet others, including Friedreich’s ataxia, are recessively inherited. A common feature is the presence of a DNA tandem repeat in the disease-associated gene and the propensity of the repeats to expand in germ and in somatic cells, with ensuing neurological and frequently also neuromuscular defects. Repeat expansion is the most frequent event in these diseases; however, sequence contractions, deletions, and mutations have also been reported. Nucleotide repeat sequences are predisposed to adopt non-B-DNA conformations, such as hairpins, cruciform, and intramolecular triple-helix structures (triplexes), also known as H-DNA. For gain-of-function disorders, oligonucleotides can be used to target either transcripts or duplex DNA and in diseases with recessive inheritance oligonucleotides may be used to alter repressive DNA or RNA conformations. Most current treatment strategies are aimed at altering transcript levels, but therapies directed against DNA are also emerging, and novel strategies targeting DNA, instead of RNA, are described. Different mechanisms using modified oligonucleotides are discussed along with the structural aspects of repeat sequences, which can influence binding modes and efficiencies." @default.
- W2944990939 created "2019-05-29" @default.
- W2944990939 creator A5036357426 @default.
- W2944990939 creator A5067890737 @default.
- W2944990939 date "2019-04-01" @default.
- W2944990939 modified "2023-10-06" @default.
- W2944990939 title "Targeted Oligonucleotides for Treating Neurodegenerative Tandem Repeat Diseases" @default.
- W2944990939 cites W1564730852 @default.
- W2944990939 cites W1686333409 @default.
- W2944990939 cites W1798173089 @default.
- W2944990939 cites W1895398290 @default.
- W2944990939 cites W1921475843 @default.
- W2944990939 cites W1952915322 @default.
- W2944990939 cites W1954017951 @default.
- W2944990939 cites W1964514885 @default.
- W2944990939 cites W1967120946 @default.
- W2944990939 cites W1970814720 @default.
- W2944990939 cites W1970986383 @default.
- W2944990939 cites W1972453061 @default.
- W2944990939 cites W1974132683 @default.
- W2944990939 cites W1975430247 @default.
- W2944990939 cites W1977983193 @default.
- W2944990939 cites W1981208686 @default.
- W2944990939 cites W1984067201 @default.
- W2944990939 cites W1985013843 @default.
- W2944990939 cites W1988744134 @default.
- W2944990939 cites W1990548509 @default.
- W2944990939 cites W1997188614 @default.
- W2944990939 cites W2000696558 @default.
- W2944990939 cites W2002644780 @default.
- W2944990939 cites W2002780348 @default.
- W2944990939 cites W200696127 @default.
- W2944990939 cites W2007578481 @default.
- W2944990939 cites W2012974517 @default.
- W2944990939 cites W2022077084 @default.
- W2944990939 cites W2025980739 @default.
- W2944990939 cites W2027385910 @default.
- W2944990939 cites W2030410903 @default.
- W2944990939 cites W2032426626 @default.
- W2944990939 cites W2035580712 @default.
- W2944990939 cites W2038332844 @default.
- W2944990939 cites W2040455172 @default.
- W2944990939 cites W2041012398 @default.
- W2944990939 cites W2044375974 @default.
- W2944990939 cites W2045636349 @default.
- W2944990939 cites W2051013729 @default.
- W2944990939 cites W2051587165 @default.
- W2944990939 cites W2053463415 @default.
- W2944990939 cites W2055234488 @default.
- W2944990939 cites W2057519110 @default.
- W2944990939 cites W2057758054 @default.
- W2944990939 cites W2059450713 @default.
- W2944990939 cites W2064808830 @default.
- W2944990939 cites W2065213545 @default.
- W2944990939 cites W2067401769 @default.
- W2944990939 cites W2071230707 @default.
- W2944990939 cites W2073115409 @default.
- W2944990939 cites W2076785565 @default.
- W2944990939 cites W2077206278 @default.
- W2944990939 cites W2080335349 @default.
- W2944990939 cites W2082501455 @default.
- W2944990939 cites W2085617522 @default.
- W2944990939 cites W2088784554 @default.
- W2944990939 cites W2089470428 @default.
- W2944990939 cites W2092574488 @default.
- W2944990939 cites W2092869314 @default.
- W2944990939 cites W2096266246 @default.
- W2944990939 cites W2103229988 @default.
- W2944990939 cites W2104979554 @default.
- W2944990939 cites W2107020608 @default.
- W2944990939 cites W2111714867 @default.
- W2944990939 cites W2121389760 @default.
- W2944990939 cites W2124161762 @default.
- W2944990939 cites W2124988362 @default.
- W2944990939 cites W2126892015 @default.
- W2944990939 cites W2130886644 @default.
- W2944990939 cites W2131173813 @default.
- W2944990939 cites W2138912547 @default.
- W2944990939 cites W2139864835 @default.
- W2944990939 cites W2143760278 @default.
- W2944990939 cites W2149919973 @default.
- W2944990939 cites W2153992384 @default.
- W2944990939 cites W2158155007 @default.
- W2944990939 cites W2168710204 @default.
- W2944990939 cites W2169345930 @default.
- W2944990939 cites W2173267258 @default.
- W2944990939 cites W2181479067 @default.
- W2944990939 cites W2188519171 @default.
- W2944990939 cites W2252602787 @default.
- W2944990939 cites W2280474488 @default.
- W2944990939 cites W2302360713 @default.
- W2944990939 cites W2304370660 @default.
- W2944990939 cites W2312976925 @default.
- W2944990939 cites W2322093705 @default.
- W2944990939 cites W2468705417 @default.
- W2944990939 cites W2520846859 @default.
- W2944990939 cites W2528282298 @default.
- W2944990939 cites W2555919336 @default.