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Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia.
Koeberl D, Schulze A, Sondheimer N, Lipshutz GS, Geberhiwot T, Li L, Saini R, Luo J, Sikirica V, Jin L, Liang M, Leuchars M, Grunewald S. Koeberl D, et al. Nature. 2024 Apr;628(8009):872-877. doi: 10.1038/s41586-024-07266-7. Epub 2024 Apr 3. Nature. 2024. PMID: 38570682 Free PMC article. Clinical Trial.
Staying on target in gene and cell therapy.
Bricker-Anthony R, Koeberl DD, Lipshutz GS, Perna F. Bricker-Anthony R, et al. Among authors: koeberl dd. Mol Ther. 2024 Sep 4;32(9):2801-2802. doi: 10.1016/j.ymthe.2024.08.010. Epub 2024 Aug 23. Mol Ther. 2024. PMID: 39178850 No abstract available.
Corrigendum to "Elevated amyloid beta peptides and total tau in cerebrospinal fluid in individuals with Creatine transporter deficiency".
Rahhal S, Farmer C, Thurm A, Wassif CA, Cawley NX, Perreault J, Dang Do A, Bianconi S, Hannah-Shmouni F, Guthrie W, Cubit LS, Miller JS, Sutton VR, Koeberl D, Porter FD. Rahhal S, et al. Among authors: koeberl d. Mol Genet Metab Rep. 2023 Oct 9;37:101013. doi: 10.1016/j.ymgmr.2023.101013. eCollection 2023 Dec. Mol Genet Metab Rep. 2023. PMID: 38053934 Free PMC article.
151 results