A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I

Our previous study delivered zinc finger nucleases to treat mice with mucopolysaccharidosis type I (MPS I), resulting in a phase I/II clinical trial (ClinicalTrials.gov: NCT02702115). However, in the clinical trial, the efficacy needs to be improved due to the low transgene expression level. To this...

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Publicat a:Mol Ther
Autors principals: Ou, Li, Przybilla, Michael J., Ahlat, Ozan, Kim, Sarah, Overn, Paula, Jarnes, Jeanine, O’Sullivan, M. Gerard, Whitley, Chester B.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2020
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7264433/
https://ncbi.nlm.nih.gov/pubmed/32278382
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.03.018
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