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Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and their associated CRISPR-associated nucleases (Cas) are among the most promising technologies for the treatment of hemoglobinopathies including Sickle Cell Disease (SCD). We are only beginning to identify the molecular variables t...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7902267/ https://www.ncbi.nlm.nih.gov/pubmed/32873924 http://dx.doi.org/10.1038/s41434-020-00192-z |