Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector.
Julie BeegleKyle HendrixHaley MacielJan A NoltaJoseph S AndersonPublished in: The journal of gene medicine (2020)
These results highlight the potential use of stem cell gene therapy as a treatment strategy for Tay-Sachs and Sandhoff disease.