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. Author manuscript; available in PMC: 2018 Jul 10.
Published in final edited form as: Cell Stem Cell. 2017 Nov 2;21(5):574–590. doi: 10.1016/j.stem.2017.10.010

Figure 1. Overview of targets for gene therapy.

Figure 1

Hematopoietic stem cells (HSCs) isolated from bone marrow can be modified ex vivo and transferred back to the recipient to produce functional, terminally-differentiated cells. Specific cellular targets and the relevant diseases and genes for gene therapy include the following: HSCs: Fanconi Anemia (FANC A–F). Platelets: Hemophilia A (Factor VIII (F8)); Hemophilia B (Factor IX (F9)); Factor X deficiency (Factor X (F10)); Wiskott-Aldrich Syndrome (Wiskott Aldrich Syndrome Protein (WASP)). Neutrophils: X-linked Chronic Granulomatous Disease (Cytochrome B-245 Beta Chain (CYBB)); Kostmann’s Syndrome (Elastase Neutrophil Expressed (ELANE)). Erythrocytes: Alpha-Thalassemia (Hemoglobin Subunit Alpha (HBA)); Beta-Thalassemia and Sickle Cell Disease (Hemoglobin Subunit Beta (HBB)); Pyruvate Kinase Deficiency (Pyruvate Kinase, Liver and RBC (PKLR)); Diamond-Blackfan Anemia (Ribosomal Protein S19 (RPS19)). Monocytes: X-linked Adrenoleukodystrophy (ATP Binding Cassette Subfamily D Member 1 (ABCD1)); Metachromatic Leukodystrophy (Arylsulfatase A (ARSA)); Gaucher disease (Glucosylceramidase Beta (GBA)); Hunter Syndrome (Iduronate 2-Sulfatase (IDS)); Mucopolysaccharidosis type I (Iduronidase, Alpha-L (IDUA)); Osteopetrosis (T-Cell Immune Regulator 1 (TCIRG1)). B Cells: Adenosine deaminase (ADA)-deficient Severe Combined Immunodeficiency (Adenosine Deaminase (ADA)); X-linked severe combined immunodeficiency (Interleukin 2 Receptor Subunit Gamma (IL2RG)); Wiskott-Aldrich Syndrome (Wiskott Aldrich Syndrome Protein (WASP)); X-linked agammaglobulinemia (Bruton’s Tyrosine Kinase (BTK)). T Cells: Adenosine Deaminase (ADA)-deficient Severe Combined Immunodeficiency (ADA); X-linked severe combined immunodeficiency (IL2RG); Wiskott-Aldrich Syndrome Protein (WASP); X-linked Hyper IgM syndrome (CD40 Ligand (CD40LG)); IPEX Syndrome (Forkhead Box P3 (FOXP3)); Early Onset Inflammatory \Disease (Interleukin 4, 10, 13 (IL-4, 10, 13));Hemophagocytic Lymphohistiocytosis (Perforin 1 (PRF1)); Cancer (Artificial T cell receptors (TCR), Cancer; Chimeric Antigen Receptor (CAR)); Human immunodeficiency virus (C-C Motif Chemokine Receptor 5 (CCR5)).