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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: karda r. 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: karda r. 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: karda r. 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.
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.
Ng J, Barral S, De La Fuente Barrigon C, Lignani G, Erdem FA, Wallings R, Privolizzi R, Rossignoli G, Alrashidi H, Heasman S, Meyer E, Ngoh A, Pope S, Karda R, Perocheau D, Baruteau J, Suff N, Antinao Diaz J, Schorge S, Vowles J, Marshall LR, Cowley SA, Sucic S, Freissmuth M, Counsell JR, Wade-Martins R, Heales SJR, Rahim AA, Bencze M, Waddington SN, Kurian MA. Ng J, et al. Among authors: karda r. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi: 10.1126/scitranslmed.aaw1564. Sci Transl Med. 2021. PMID: 34011628 Free PMC article.
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: karda r. 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.
Continual Conscious Bioluminescent Imaging in Freely Moving Mice.
Diaz JA, Geard A, FitzPatrick LM, Delhove JMKM, Buckley SMK, Waddington SN, McKay TR, Karda R. Diaz JA, et al. Among authors: karda r. Methods Mol Biol. 2020;2081:161-175. doi: 10.1007/978-1-4939-9940-8_11. Methods Mol Biol. 2020. PMID: 31721124
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