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Getting the Most: Enhancing Efficacy by Promoting Erythropoiesis and Thrombopoiesis after Gene Therapy in Mice with Hurler Syndrome

Novel strategies are needed to solve the conundrum of achieving clinical efficacy with high vector copy numbers (VCNs) in hematopoietic stem cells (HSCs) while attempting to minimize the potential risk of oncogenesis in lentiviral vector (LV)-mediated gene therapy clinical trials. We previously repo...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Han, Jing-fen, El-Amouri, Salim S., Dai, Mei, Cao, Phuong, Pan, Dao
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6205327/
https://ncbi.nlm.nih.gov/pubmed/30397627
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.10.001
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