title: Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs.
abstract: Mutations causing X-linked severe combined immunodeficiency (SCID-X1) reduce immune cell populations and function and may be amenable to targeted gene correction strategies. Now in Cell Stem Cell, Menon et al. (2015) correct SCID-X1-related blood differentiation defects by TALEN-mediated genome editing in patient-derived iPSCs, suggesting a possible strategy for autologous cell therapy of SCID-X1.
authors.affiliation: San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, 20132 Milan, Italy. Electronic address: biffi.alessandra@hsr.it.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Biffi
mesh_terms: D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D016219:Immunotherapy, Adoptive; D057026:Induced Pluripotent Stem Cells / Q000502:physiology*; D007694:Killer Cells, Natural / Q000502:physiology*; D054504:Precursor Cells, T-Lymphoid / Q000502:physiology*; D012038:Regeneration; D044968:Regenerative Medicine; D053632:X-Linked Combined Immunodeficiency Diseases / Q000628:therapy*