An Innovative Hematopoietic Stem Cell Gene Therapy Approach Benefits CLN1 Disease in the Mouse Model

[Nature Communications] TAMARIN Phase-II, multicenter, single-arm clinical trial assessed tamoxifen’s safety and activity in patients with stable myeloproliferative neoplasms, no prior thrombotic events and mutated JAK2V617F, CALRins5 or CALRdel52 peripheral blood allele burden ≥20%.

[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.