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343 results

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Page 1
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: diaz ja. Science. 2024 Oct 4;386(6717):61-69. doi: 10.1126/science.adk2539. Epub 2024 Oct 3. Science. 2024. PMID: 39361759 Free PMC article.
Creation of de novo cryptic splicing for ALS/FTD precision medicine.
Wilkins OG, Chien MZYJ, Wlaschin JJ, Pisliakova M, Thompson D, Digby H, Simkin RL, Diaz JA, Mehta PR, Keuss MJ, Zanovello M, Brown AL, Harley P, Darbey A, Karda R, Fisher EMC, Cunningham TJ, Le Pichon CE, Ule J, Fratta P. Wilkins OG, et al. Among authors: diaz ja. bioRxiv [Preprint]. 2023 Nov 15:2023.11.15.565967. doi: 10.1101/2023.11.15.565967. bioRxiv. 2023. Update in: Science. 2024 Oct 4;386(6717):61-69. doi: 10.1126/science.adk2539. PMID: 38014203 Free PMC article. Updated. Preprint.
Genetic therapeutic advancements for Dravet Syndrome.
Chilcott E, Díaz JA, Bertram C, Berti M, Karda R. Chilcott E, et al. Among authors: diaz ja. Epilepsy Behav. 2022 Jul;132:108741. doi: 10.1016/j.yebeh.2022.108741. Epub 2022 May 30. Epilepsy Behav. 2022. PMID: 35653814 Free article. Review.
Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation.
Counsell JR, De Brabandere G, Karda R, Moore M, Greco A, Bray A, Diaz JA, Perocheau DP, Mock U, Waddington SN. Counsell JR, et al. Among authors: diaz ja. Mol Ther Methods Clin Dev. 2020 Dec 15;20:357-365. doi: 10.1016/j.omtm.2020.12.005. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33553484 Free PMC article.
Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.
Counsell JR, Karda R, Diaz JA, Carey L, Wiktorowicz T, Buckley SMK, Ameri S, Ng J, Baruteau J, Almeida F, de Silva R, Simone R, Lugarà E, Lignani G, Lindemann D, Rethwilm A, Rahim AA, Waddington SN, Howe SJ. Counsell JR, et al. Among authors: diaz ja. Mol Ther Nucleic Acids. 2018 Sep 7;12:626-634. doi: 10.1016/j.omtn.2018.07.006. Epub 2018 Aug 3. Mol Ther Nucleic Acids. 2018. PMID: 30081233 Free PMC article.
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer.
Baruteau J, Perocheau DP, Hanley J, Lorvellec M, Rocha-Ferreira E, Karda R, Ng J, Suff N, Diaz JA, Rahim AA, Hughes MP, Banushi B, Prunty H, Hristova M, Ridout DA, Virasami A, Heales S, Howe SJ, Buckley SMK, Mills PB, Gissen P, Waddington SN. Baruteau J, et al. Among authors: diaz ja. Nat Commun. 2018 Aug 29;9(1):3505. doi: 10.1038/s41467-018-05972-1. Nat Commun. 2018. PMID: 30158522 Free PMC article.
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.
Karda R, Rahim AA, Wong AMS, Suff N, Diaz JA, Perocheau DP, Tijani M, Ng J, Baruteau J, Martin NP, Hughes M, Delhove JMKM, Counsell JR, Cooper JD, Henckaerts E, Mckay TR, Buckley SMK, Waddington SN. Karda R, et al. Among authors: diaz ja. Sci Rep. 2020 Feb 7;10(1):2121. doi: 10.1038/s41598-020-59075-3. Sci Rep. 2020. PMID: 32034258 Free PMC article.
343 results