Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

2018-04-11 07:22:12 | BioPortfolio


This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant


Pediatric patients with mucopolysaccharidosis type I will be treated with genetically modified autologous hematopoietic stem cells collected from mobilized peripheral blood (or bone marrow if mobilization is not feasible) and transduced with IDUA lentiviral vector encoding for the human α-L-iduronidase gene.

Patients will be followed for 1 year after gene therapy; after 1 year follow-up they will be asked to enroll in a long term study in order to monitor the safety and efficacy of the gene therapy treatment for up to 8 years.

Study Design


Mucopolysaccharidosis IH


Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.


Ospedale San Raffaele


Not yet recruiting


IRCCS San Raffaele

Results (where available)

View Results


Published on BioPortfolio: 2018-04-11T07:22:12-0400

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Medical and Biotech [MESH] Definitions

Glycoproteins found on immature hematopoietic cells and endothelial cells. They are the only molecules to date whose expression within the blood system is restricted to a small number of progenitor cells in the bone marrow.

Stem cells derived from HEMATOPOIETIC STEM CELLS. Derived from these myeloid progenitor cells are the MEGAKARYOCYTES; ERYTHROID CELLS; MYELOID CELLS; and some DENDRITIC CELLS.

Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms.

A member of the prominin family, AC133 Antigen is a 5-transmembrane antigen occurring as several isoforms produced by alternative splicing which are processed into mature forms. In humans, it is expressed as a subset of CD34 (bright) human hematopoietic stem cells and CD34 positive leukemias. Functionally, it is associated with roles in cell differentiation, proliferation, and apoptosis. Specifically, it regulates the organization of apical plasma membrane in epithelial cells, disk morphogenesis during early retinal development, MAPK and Akt signaling pathways, and in cholesterol metabolism.

Progenitor cells from which all blood cells derive.

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