Overview

• Scientists from SR-Tiget and CIEMAT/CIBERER discovered that high numbers of circulating hematopoietic stem and progenitor cells in newborn mice enable direct in vivo lentiviral gene transfer.
• In mouse models of ADA-SCID, autosomal recessive osteopetrosis and Fanconi anemia, postnatal gene delivery achieved significant therapeutic benefits, including life extension and bone marrow recovery.
• Use of clinically approved mobilizer drugs G-CSF and Plerixafor boosted circulating stem cell counts, extending the effective treatment window and improving gene transfer efficiency.
• Circulating hematopoietic stem and progenitor cells were also detected in the blood of human newborns, indicating that the neonatal window may exist in infants.
• Further preclinical studies are planned to refine vector design, assess safety and advance toward initial clinical testing for monogenic blood disorders in newborns.