PMC Articles for MedGen (Select 869388) - PMC

Result Filters

Links from MedGen

Items: 4

Select item 106806181.

AAV-mediated gene therapy for Sialidosis

Diantha van de Vlekkert, Huimin Hu, Leigh E. Fremuth, Scott A. Brown, Jason A. Weesner, Elida Gomero, Yvan Campos, Alessandra d’Azzo

Version 1. bioRxiv. Preprint. 2023 Nov 13. doi: 10.1101/2023.11.10.566667

Published in:: Mol Ther. 2024 Jul 3; 32(7): 2094–2112.

PMCID:: PMC10680618

Abstract Article

Select item 86406472.

AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study

Huimin Hu, Rosario Mosca, Elida Gomero, Diantha van de Vlekkert, Yvan Campos, Leigh E. Fremuth, Scott A. Brown, Jason A. Weesner, Ida Annunziata, Alessandra d’Azzo

Mol Ther Methods Clin Dev. 2021 Dec 10; 23: 644–658. Published online 2021 Oct 28. doi: 10.1016/j.omtm.2021.10.007

PMCID:: PMC8640647

Abstract Article PDF–7.9M

Select item 71413193.

Conventional and Unconventional Therapeutic Strategies for Sialidosis Type I

Rosario Mosca, Diantha van de Vlekkert, Yvan Campos, Leigh E. Fremuth, Jaclyn Cadaoas, Vish Koppaka, Emil Kakkis, Cynthia Tifft, Camilo Toro, Simona Allievi, Cinzia Gellera, Laura Canafoglia, Gepke Visser, Ida Annunziata, Alessandra d’Azzo

J Clin Med. 2020 Mar; 9(3): 695. Published online 2020 Mar 4. doi: 10.3390/jcm9030695