Prospects of Stem Cell Therapy for Autism Spectrum Disorders

Xuejun Kong, MD, Xiaochun Wang, PhD, William Stone, PhD


Autism is a group of highly complicated neurodevelopment disorders affecting 1 in every 110 children born in USA. The etiology and pathophysiology are poorly understood and it currently has no universally accepted therapy or cure. Stem cell replacement therapy via transplantation potentially reverse brain hypo perfusion and immune dysfunction, which are considered to be two major pathophysiology mechanisms of autism. Recent advances in the in vitro study of disease modeling of autism reveals disease-specific cellular defects and reversible symptoms, opens the possibility of cell therapy and offers new hopes for cures. However, this new therapy is still in its infancy. There are many technological barrier such as immune-rejection, tissue migration and integration, cancer risk, safety concern, proper development in the inner environment and clinical barriers of individual sensitivity and liability, disease complexity, and more, which will need to be overcome before reaching new era of therapy. It will be necessary to conduct in vivo animal studies and then further clinical trials, however, before any potential clinical application in autism and other human disease can be realized.


stem cell, stem cell therapy, autism

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