Antisense oligonucleotides correct the familial dysautonomia splicing defect in IKBKAP transgenic mice
Sinha R, Kim YJ, Nomakuchi T, Sahashi K, Hua Y, Rigo F, Bennett CF, Krainer AR. Nucleic Acids Res. 2018 Jun 1;46(10):4833-4844. The three joint first authors: Rahul Sinha Young Jin Kim Tomoki Nomakuchi Currently, the most successful oligonucleotide ...