NHEJ介导的CRISPR-Cas9诱导的DNA断裂修复有效纠正范科尼贫血患者HSPCs的突变。

F. Román-Rodríguez, Laura Ugalde, L. Álvarez, B. Díez, M. Ramírez, Cristina Risueño, M. Cortón, M. Bogliolo, S. Bernal, Francesc March, C. Ayuso, H. Hanenberg, J. Sevilla, S. Rodríguez-Perales, R. Torres-Ruiz, J. Surrallés, J. Bueren, P. Río
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NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia.
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