Rescuing the cytolytic function of APDS1 patient T cells via TALEN-mediated PIK3CD gene correction.

"Declaration of interests S.L., A.B., A.J., A.M., P.D., and J.V. are Cellectis employees. TALEN is a Cellectis patented technology. M.C. has consulted for Cellectis. S.K. reports collaboration agreements and payments from Cellectis. J.V., P.D., A.J., L.P., M.C., and S.K. are inventors on an EP patent application related to this work."

"S.K. is a Centre National de la Recherche Scientifique staff researcher. We thank the CRB-DNA from Necker for the establishment of immortalized B-EBV; the Etablissement Français du Sang for blood supply from healthy donors; the cytometry platform from Necker; the Human Lymphohematopoiesis laboratory for discussion. The LabTech Single-Cell@Imagine, was supported by the Paris Region and the “Investissement d’avenir” program through 2019 ATF funding—Sésame Filières PIA (grant 3877871). Cellectis and Imagine would like to thank the generous contribution of all the patients involved in this study. Some schemes were created with biorender.com."