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Gene therapy by allele selection in a mouse model of beta-thalassemia
To be of therapeutic use, autologous stem cells derived from patients with inherited genetic disorders require genetic modification via gene repair or insertion. Here, we present proof of principle that, for diseases associated with dominant alleles (gain-of-function or haploinsufficient loss-of-fun...
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| Main Authors: | , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society for Clinical Investigation
2011
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3026741/ https://ncbi.nlm.nih.gov/pubmed/21293060 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI45377 |
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