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 Apr 17. PubMed
Targeting Amyloid-β Precursor Protein, APP, Splicing with Antisense Oligonucleotides Reduces Toxic Amyloid-β Production
Chang JL, Hinrich AJ, Roman B, Norrbom M, Rigo F, Marr RA, Norstrom EM, Hastings ML. MolTher. 2018 Mar 6. pii: S1525-0016(18)30105-9. PubMed
The aberrant splicing of BAF45d links splicing regulation and transcription in glioblastoma
Aldave G, Gonzalez-Huarriz M, Rubio A, Romero JP, Ravi D, Miñana B, Cuadrado-Tejedor M, GarcĂa-Osta A, Verhaak R, Xipell E, Martinez-VĂ©lez N, Acanda de la Rocha A, Puigdelloses M, GarcĂa-Moure M, Marigil M, GĂ¡llego PĂ©rez-Larraya J, MarĂn-Bejar O, Huarte M, Carro MS, Ferrarese R, Belda-Iniesta ...