title: Genetic modification of hematopoietic stem cells: recent advances in the gene therapy of inherited diseases.
abstract: Hematopoietic stem cells constitute a rare population of precursor cells with remarkable properties for being used as targets in gene therapy protocols. The last years have been particularly productive both in the fields of gene therapy and stem cell biology. Results from ongoing clinical trials have shown the first unquestionable clinical benefits of immunodeficient patients transplanted with genetically modified autologous stem cells. On the other hand, severe side effects in a few patients treated with gene therapy have also been reported, indicating the usefulness of further improving the vectors currently used in gene therapy clinical trials. In the field of stem cell biology, evidence showing the plastic potential of adult hematopoietic stem cells and data indicating the multipotency of adult mesenchymal precursor cells have been presented. Also, the generation of embryonic stem cells by means of nuclear transfer techniques has appeared as a new methodology with direct implications in gene therapy.
authors.affiliation: Hematopoietic Gene Therapy Program, Comisión Interministerial de Ciencia y Tecnología/Fundación Marcelino Botín, Madrid, Spain. juan.bueren@ciemat.es
authors.forename: Juan A
authors.initials: JA
authors.lastname: Bueren
authors.forename: Guillermo
authors.initials: G
authors.lastname: Guenechea
authors.forename: José A
authors.initials: JA
authors.lastname: Casado
authors.forename: María Luisa
authors.initials: ML
authors.lastname: Lamana
authors.forename: José C
authors.initials: JC
authors.lastname: Segovia
mesh_terms: D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006402:Hematologic Diseases / Q000151:congenital*; D006412:Hematopoietic Stem Cells / Q000502:physiology*; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics / Q000628:therapy