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...

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Detalhes bibliográficos
Publicado no:Gene Ther
Main Authors: Modarai, Shirin R., Kanda, Sambee, Bloh, Kevin, Opdenaker, Lynn M., Kmiec, Eric B.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2020
Assuntos:
Brief Communication
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7902267/
https://ncbi.nlm.nih.gov/pubmed/32873924
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-020-00192-z
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