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Perforin Gene Transfer Into Hematopoietic Stem Cells Improves Immune Dysregulation in Murine Models of Perforin Deficiency.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 4, p. 737, doi. 10.1038/mt.2014.242
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- Publication type:
- Article
The β-Globin Locus Control Region in Combination With the EF1α Short Promoter Allows Enhanced Lentiviral Vector-mediated Erythroid Gene Expression With Conserved Multilineage Activity.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 7, p. 1400, doi. 10.1038/mt.2012.50
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- Publication type:
- Article
Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates.
- Published in:
- FASEB Journal, 2015, v. 29, n. 9, p. 3876, doi. 10.1096/fj.14-269092
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- Article
Rescue of Pyruvate Kinase Deficiency in Mice by Gene Therapy Using the Human Isoenzyme.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 12, p. 2000, doi. 10.1038/mt.2009.200
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- Article