Preclinical Assessment for Gene Addition Strategy in Familial Hemophagocytic Lymphohistiocytosis Related to Munc 13-4 Deficiency

Lagresle-Peyrou, C; Diana, JS; Magrin, E; Aussel, C; Sorel, N; Aslan, Y; Roudaut, C; Schwartz, E; Gabrion, A; Tuchmann-Durand, C; Lefrere, F; Galy, A; Moshous, D; Cavazzana, M

BLOOD, 2023; 142 ():