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2019 4
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13 results

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Terms must have at least 4 characters before the first wildcard *. Wildcard ignored: rw*
Page 1
Delivery of oligonucleotide-based therapeutics: challenges and opportunities.
Hammond SM, Aartsma-Rus A, Alves S, Borgos SE, Buijsen RAM, Collin RWJ, Covello G, Denti MA, Desviat LR, Echevarría L, Foged C, Gaina G, Garanto A, Goyenvalle AT, Guzowska M, Holodnuka I, Jones DR, Krause S, Lehto T, Montolio M, Van Roon-Mom W, Arechavala-Gomeza V. Hammond SM, et al. EMBO Mol Med. 2021 Apr 9;13(4):e13243. doi: 10.15252/emmm.202013243. Epub 2021 Apr 6. EMBO Mol Med. 2021. PMID: 33821570 Free PMC article. Review.
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial.
Russell SR, Drack AV, Cideciyan AV, Jacobson SG, Leroy BP, Van Cauwenbergh C, Ho AC, Dumitrescu AV, Han IC, Martin M, Pfeifer WL, Sohn EH, Walshire J, Garafalo AV, Krishnan AK, Powers CA, Sumaroka A, Roman AJ, Vanhonsebrouck E, Jones E, Nerinckx F, De Zaeytijd J, Collin RWJ, Hoyng C, Adamson P, Cheetham ME, Schwartz MR, den Hollander W, Asmus F, Platenburg G, Rodman D, Girach A. Russell SR, et al. Nat Med. 2022 May;28(5):1014-1021. doi: 10.1038/s41591-022-01755-w. Epub 2022 Apr 4. Nat Med. 2022. PMID: 35379979 Free PMC article. Clinical Trial.
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4.
Vázquez-Domínguez I, Öktem M, Winkelaar FA, Nguyen TH, Hoogendoorn ADM, Roschi E, Astuti GDN, Timmermans R, Suárez-Herrera N, Bruno I, Ruiz-Llombart A, Brealey J, de Jong OG, Collin RWJ, Mastrobattista E, Garanto A. Vázquez-Domínguez I, et al. Mol Ther Nucleic Acids. 2024 Sep 26;35(4):102345. doi: 10.1016/j.omtn.2024.102345. eCollection 2024 Dec 10. Mol Ther Nucleic Acids. 2024. PMID: 39494150 Free PMC article.
Superparamagnetic Nanoparticle Delivery to the Cochlea Through Round Window by External Magnetic Field: Feasibility and Toxicity.
Leterme G, Guigou C, Oudot A, Collin B, Boudon J, Millot N, Geissler A, Belharet K, Bozorg Grayeli A. Leterme G, et al. Surg Innov. 2019 Dec;26(6):646-655. doi: 10.1177/1553350619867217. Epub 2019 Sep 3. Surg Innov. 2019. PMID: 31478462
The objective of this study was to evaluate the feasibility and toxicity of superparamagnetic iron oxide nanoparticles (SPIONs) administered into the cochlea through the round window (RW) by an external magnetic field. Materials and Methods. In 5 Wistar rats, the left R …
The objective of this study was to evaluate the feasibility and toxicity of superparamagnetic iron oxide nanoparticles (SPIONs) administered …
13 results