Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
Gee P, Lung MSY, Okuzaki Y, Sasakawa N, Iguchi T, Makita Y, Hozumi H, Miura Y, Yang LF, Iwasaki M, Wang XH, Waller MA, Shirai N, Abe YO, Fujita Y, Watanabe K, Kagita A, Iwabuchi KA, Yasuda M, Xu H, Noda T, Komano J, Sakurai ...
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Neurology. 2020 Mar 5. PubMed
Splice switching an oncogenic ratio of SmgGDS isoforms as a strategy to diminish malignancy
Brandt AC, McNally L, Lorimer EL, Unger B, Koehn OJ, Suazo KF, Rein L, Szabo A, Tsaih SW, Distefano MD, Flister MJ, Rigo F, McNally MT, Williams CL. Proc Natl Acad Sci U S A. 2020 Feb 18;117(7):3627-3636. PubMed
Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model
Sheng L, Rigo F, Bennett CF, Krainer AR, Hua Y. Nucleic Acids Res. 2020 Feb 27. PubMed
Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy
Zhou H, Meng J, Malerba A, Catapano F, Sintusek P, Jarmin S, Feng L, Lu-Nguyen N, Sun L, Mariot V, Dumonceaux J, Morgan JE, Gissen P, Dickson G, Muntoni F. J Cachexia Sarcopenia Muscle. 2020 Feb 7. PubMed
RNA Splicing by the Spliceosome.
Wilkinson ME, Charenton C, Nagai K. Annu Rev Biochem. 2019 Dec 3. PubMed
Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides.
Scharner J, Ma WK, Zhang Q, Lin KT, Rigo F, Bennett CF, Krainer AR. Nucleic Acids Res. 2019 Dec 5. PubMed
Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy.
Aupy P, EchevarrÃa L, Relizani K, Zarrouki F, Haeberli A, Komisarski M, Tensorer T, Jouvion G, Svinartchouk F, Garcia L, Goyenvalle A. Mol Ther Nucleic Acids. 2019 Nov 27;19:371-383. PubMed
The changing paradigm of intron retention: regulation, ramifications and recipes.
Monteuuis G, Wong JJL, Bailey CG, Schmitz U, Rasko JEJ. Nucleic Acids Res. 2019 Nov 14. PubMed
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine ...
