Unbiased assessment of genome integrity and purging of adverse outcomes at the target locus upon editing of CD4+ T-cells for the treatment of Hyper IgM1.
Canarutto D, Asperti C, Vavassori V, Porcellini S, Rovelli E, Paulis M, Ferrari S, Varesi A, Fiumara M, Jacob A, Sergi Sergi L, Visigalli I, Ferrua F, González-Granado LI, Lougaris V, Finocchi A, Villa A, Radrizzani M, Naldini L.
Canarutto D, et al.
EMBO J. 2023 Dec 1;42(23):e114188. doi: 10.15252/embj.2023114188. Epub 2023 Nov 2.
EMBO J. 2023.
PMID: 37916874
Free PMC article.
Contrary to established gene therapies, there is limited data on the genomic alterations following long-range gene editing, and no consensus on the relevant assays. We developed drop-off digital PCR assays for unbiased detection of large on-target deletions and found them …
Contrary to established gene therapies, there is limited data on the genomic alterations following long-range gene editing, and no consensus …