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Loss of TDP-43 induces synaptic dysfunction that is rescued by UNC13A splice-switching ASOs.
Keuss MJ, Harley P, Ryadnov E, Jackson RE, Zanovello M, Wilkins OG, Barattucci S, Mehta PR, Oliveira MG, Parkes JE, Sinha A, Correa-Sánchez AF, Oliver PL, Fisher EMC, Schiavo G, Shah M, Burrone J, Fratta P. Keuss MJ, et al. Among authors: harley p. bioRxiv [Preprint]. 2024 Jun 24:2024.06.20.599684. doi: 10.1101/2024.06.20.599684. bioRxiv. 2024. PMID: 38979232 Free PMC article. Preprint.
Creation of de novo cryptic splicing for ALS and FTD precision medicine.
Wilkins OG, Chien MZYJ, Wlaschin JJ, Barattucci S, Harley P, Mattedi F, Mehta PR, Pisliakova M, Ryadnov E, Keuss MJ, Thompson D, Digby H, Knez L, Simkin RL, Diaz JA, Zanovello M, Brown AL, Darbey A, Karda R, Fisher EMC, Cunningham TJ, Le Pichon CE, Ule J, Fratta P. Wilkins OG, et al. Among authors: harley p. Science. 2024 Oct 4;386(6717):61-69. doi: 10.1126/science.adk2539. Epub 2024 Oct 3. Science. 2024. PMID: 39361759 Free PMC article.
Modelling renal defects in Bardet-Biedl syndrome patients using human iPS cells.
Williams J, Hurling C, Munir S, Harley P, Machado CB, Cujba AM, Alvarez-Fallas M, Danovi D, Lieberam I, Sancho R, Beales P, Watt FM. Williams J, et al. Among authors: harley p. Front Cell Dev Biol. 2023 Jun 2;11:1163825. doi: 10.3389/fcell.2023.1163825. eCollection 2023. Front Cell Dev Biol. 2023. PMID: 37333983 Free PMC article.
Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections.
Paredes-Redondo A, Harley P, Maniati E, Ryan D, Louzada S, Meng J, Kowala A, Fu B, Yang F, Liu P, Marino S, Pourquié O, Muntoni F, Wang J, Lieberam I, Lin YY. Paredes-Redondo A, et al. Among authors: harley p. Sci Adv. 2021 Sep 10;7(37):eabi8787. doi: 10.1126/sciadv.abi8787. Epub 2021 Sep 10. Sci Adv. 2021. PMID: 34516770 Free PMC article.
65 results