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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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Detalles Bibliográficos
Autores principales: Han, Jing-fen, El-Amouri, Salim S., Dai, Mei, Cao, Phuong, Pan, Dao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6205327/
https://www.ncbi.nlm.nih.gov/pubmed/30397627
http://dx.doi.org/10.1016/j.omtm.2018.10.001