Libmeldy is a gene therapy that compensates for the lack of ARSA by introducing a correct copy of the gene into the genome of blood stem cells. To do this, the body’s own CD34-positive hematopoietic stem cells (HSC) are isolated from the patient’s peripheral blood or bone marrow and then modified by inserting copies of the ARSA gene into their genome using a lentiviral vector. These modified cells are reinfused into the patient once. In the body, the stem cells colonize the bone marrow and form new blood cells. These supply all tissues with the previously missing enzyme. According to the technical information, it is assumed that the effect of the gene therapy will persist over the long term. The longest follow-up period achieved so far is eight years.
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