Evaluation of gene expression regulation and function after CRISPR/Cas9-based genome editing strategies for the treatment of RAG1 defects

C. Brandas, S. Porcellini, M. C. Castiello,S. Ferrari, V. Vavassori,D. Canarutto,M. Paulis, D. Strina,I. Merelli, P. Genovese,L. D. Notarangelo,L. Naldini, A. Villa

HUMAN GENE THERAPY(2022)

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