In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery. [electronic resource]
Producer: 20180905Description: 13304 p. digitalISSN:- 2041-1723
- Animals
- Cell Line
- DNA -- administration & dosage
- Disease Models, Animal
- Gene Editing -- methods
- Genetic Therapy -- methods
- Hematopoietic Stem Cells -- metabolism
- Hemoglobins -- analysis
- Humans
- Injections, Intravenous
- Mice
- Mice, Transgenic
- Nanoparticles -- administration & dosage
- Peptide Nucleic Acids -- administration & dosage
- Proto-Oncogene Proteins c-kit -- metabolism
- Stem Cell Factor -- administration & dosage
- beta-Globins -- genetics
- beta-Thalassemia -- blood
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Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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