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Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases. [PDF]

Genes (Basel)
Wilton-Clark H, Yan E, Yokota T, Yokota T.   +3 more
europepmc   +1 more source

Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing. [PDF]

Mol Ther Nucleic Acids
Aguti S, Cheng S, Ala P, Briggs S, Muntoni F, Zhou H.   +5 more
europepmc   +1 more source

Correction: Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome.

Elife
Lee D, Chen W, Kaku HN, Zhuo X, Chao ES, Soriano A, Kuncheria A, Flores S, Kim JH, Rivera A, Rigo F, Jafar-Nejad P, Beaudet AL, Caudill MS, Xue M.   +14 more
europepmc   +1 more source

The diversity ofSNCAtranscripts in neurons, and its impact on antisense oligonucleotide therapeutics

Evans JR, Gustavsson EK, Doykov I, Murphy D, Virdi GS, Lachica J, Röntgen A, Murtada MH, Pang CW, Macpherson H, Wernick AI, Toomey CE, Athauda D, Choi ML, Hardy J, Wood NW, Vendruscolo M, Mills K, Heywood W, Ryten M, Gandhi S.   +20 more
europepmc   +1 more source

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Antisense Oligonucleotides

Nephron Experimental Nephrology, 1998
Antisense technology was developed to inhibit gene expression by utilizing an oligonucleotide complementary to the mRNA which encodes the target gene. There are a few possible mechanisms for the inhibitory effects of antisense oligonucleotides. Among them, degradation of mRNA by RNase H is considered to be the major mechanism of action for antisense ...
N, Kashihara, Y, Maeshima, H, Makino
openaire   +2 more sources