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mRNA trans-splicing in gene therapy for genetic diseases.
Berger A, Maire S, Gaillard MC, Sahel JA, Hantraye P, Bemelmans AP. Berger A, et al. Among authors: bemelmans ap. Wiley Interdiscip Rev RNA. 2016 Jul;7(4):487-98. doi: 10.1002/wrna.1347. Epub 2016 Mar 28. Wiley Interdiscip Rev RNA. 2016. PMID: 27018401 Free PMC article. Review.
Fine-tuning FAM161A gene augmentation therapy to restore retinal function.
Arsenijevic Y, Chang N, Mercey O, El Fersioui Y, Koskiniemi-Kuendig H, Joubert C, Bemelmans AP, Rivolta C, Banin E, Sharon D, Guichard P, Hamel V, Kostic C. Arsenijevic Y, et al. Among authors: bemelmans ap. EMBO Mol Med. 2024 Apr;16(4):805-822. doi: 10.1038/s44321-024-00053-x. Epub 2024 Mar 19. EMBO Mol Med. 2024. PMID: 38504136 Free PMC article.
The C-Terminal Domain of LRRK2 with the G2019S Substitution Increases Mutant A53T α-Synuclein Toxicity in Dopaminergic Neurons In Vivo.
Cresto N, Gardier C, Gaillard MC, Gubinelli F, Roost P, Molina D, Josephine C, Dufour N, Auregan G, Guillermier M, Bernier S, Jan C, Gipchtein P, Hantraye P, Chartier-Harlin MC, Bonvento G, Van Camp N, Taymans JM, Cambon K, Liot G, Bemelmans AP, Brouillet E. Cresto N, et al. Among authors: bemelmans ap. Int J Mol Sci. 2021 Jun 23;22(13):6760. doi: 10.3390/ijms22136760. Int J Mol Sci. 2021. PMID: 34201785 Free PMC article.
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