An Innovative Hematopoietic Stem Cell Gene Therapy Approach Benefits CLN1 Disease in the Mouse Model
Scientists provided evidence of the efficacy and feasibility of a novel approach to treat CLN1 disease and possibly other neurodegenerative conditions, paving the way for its future clinical application.
[Haematologica] 301 patients with a median age 60 years were enrolled. Of them, 287 and 274 patients collected ≥2 and ≥4×106 CD34+ cells/kg, respectively, with a median of 9.9×106 CD34+ cells/kg collected.
[EMBO Journal] The authors provide an in-depth review of the metabolic and epigenetic interplay regulating hematopoietic stem cell fate, and discuss the influence of metabolic stress stimuli, as well as alterations occurring during leukemic development.