Cellectis publishes new research paper in Molecular Therapy

Cellectis announced the publication of a new research paper in Molecular Therapy, demonstrating that TALEN-mediated intron editing of hematopoietic stem and progenitor cells, or HSPCs, enables transgene expression restricted to the myeloid lineage. This approach could unlock new therapeutic avenues for the treatment of inborn metabolic diseases as well as neurological diseases that require delivery of therapeutics to the brain. The CD11b intron-specific gene insertion approach efficiently restricts the expression of a desired transgene to the myeloid lineage, preventing its overexpression by stem cells or by other differentiated lineages. The insertion of an IDUA transgene in the first intron of the CD11b gene enables to express IDUA (the enzyme missing in Mucopolysaccharidosis type I patients), in a myeloid-specific manner without affecting CD11b endogenous expression. Edited HSPC exhibited robust engraftment in the bone marrow of immunodeficient mice, displayed multi-lineage differentiation in various hematopoietic tissues and showed significant presence in the brain as myeloid cells.