Allogeneic stem cells can be administered multiple times over the course of days in uniform dosages, they are immune system privileged, cell rejection is not an issue and Human Leukocyte Antigen (HLA) matching is not necessary.
No need to collect stem cells from the patient’s hip bone or fat under anesthesia, which especially for small children and their parents, can be an unpleasant ordeal.
No need to administer chemotherapy drugs like granulocyte-colony stimulating factor (G-CSF or GCSF) to stimulate the bone marrow to produce granulocytes and stem cells and release them into the bloodstream.
Regardless of the type of genetic mutation, Wharton’s jelly derived mesenchymal stem cell administration appears to be an effective and safe option. There are no serious adverse events or ophthalmic/systemic side effects for 6 months follow-up.