Treatment of inherited neurometabolic diseases: the future.
The past 10 years' experience with bone marrow transplantation from normal, immunologically compatible donors indicates its possible use in various neurometabolic diseases, particularly in a patient who has not suffered irreparable brain damage. This experience may be a prelude to treatment by somatic gene therapy. This can be applied as an autologous bone marrow transplant, grafting the patient's own stem cells inserted with the normal gene. Although somatic gene therapy will be relatively easy for tissues with dividing cells, its application to target tissues with little or no cell division may pose difficulties. Meanwhile, techniques for the preservation, culture, and grafting of fetal neurons in humans have been developed and have been used in the treatment of Parkinson's disease. These procedures could readily be transferred to the treatment of other neurodegenerative diseases that cause significant morbidity, but ethical, legal, and religious considerations must be taken into account. All these efforts promise novel and improved management of inborn neurometabolic errors.
Ozand
P T
PT
Department of Pediatrics, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.
Gascon
G G
GG
D000818:Animals; D016026:Bone Marrow Transplantation; D001928:Brain Diseases, Metabolic / Q000235:genetics / Q000628:therapy*; D016380:Brain Tissue Transplantation; D016332:Fetal Tissue Transplantation; D005544:Forecasting; D015316:Genetic Therapy; D006801:Humans; D007223:Infant; D008661:Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy*