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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...
Autores principales: | Han, Jing-fen, El-Amouri, Salim S., Dai, Mei, Cao, Phuong, Pan, Dao |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2018
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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 |
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