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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

Summary

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

Description

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

Conditions

Mucopolysaccharidosis IH

Intervention

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.

Location

Ospedale San Raffaele
Milano
Italy
20132

Status

Not yet recruiting

Source

IRCCS San Raffaele

Results (where available)

View Results

Links

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

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Medical and Biotech [MESH] Definitions

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Progenitor cells from which all blood cells derive.

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