A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2

In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.GALGT2 (also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular dystrophy using intravascular limb infusion. Subject 1 (age 8.9 years at dosing) received 2.5 × 10(13) vector genome (vg)/kg per leg (5 × 10(1...

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Detalles Bibliográficos
Autores principales: Flanigan, Kevin M., Vetter, Tatyana A., Simmons, Tabatha R., Iammarino, Megan, Frair, Emma C., Rinaldi, Federica, Chicoine, Louis G., Harris, Johan, Cheatham, John P., Cheatham, Sharon L., Boe, Brian, Waldrop, Megan A., Zygmunt, Deborah A., Packer, Davin, Martin, Paul T.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9483573/
https://www.ncbi.nlm.nih.gov/pubmed/36186954
http://dx.doi.org/10.1016/j.omtm.2022.08.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9483573/
https://www.ncbi.nlm.nih.gov/pubmed/36186954
http://dx.doi.org/10.1016/j.omtm.2022.08.009

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