An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
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Publisher
United States: Elsevier Inc
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Language
English
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Publisher
United States: Elsevier Inc
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Scope and Contents
Contents
Autologous transplantation of hematopoietic stem cells transduced with a lentiviral vector (LV) expressing an anti-sickling HBB variant is a potential treatment for sickle cell disease (SCD). With a clinical trial as our ultimate goal, we generated LV constructs containing an anti-sickling HBB transgene (HBBAS3), a minimal HBB promoter, and differe...
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Full title
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6105766
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6105766
Other Identifiers
ISSN
2329-0501
E-ISSN
2329-0501
DOI
10.1016/j.omtm.2018.07.012
