Stem Cell Technology for (Epi)genetic Brain Disorders

Advances in Experimental Medicine and Biology
Renzo J M RiemensRaul Delgado-Morales

Abstract

Despite the enormous efforts of the scientific community over the years, effective therapeutics for many (epi)genetic brain disorders remain unidentified. The common and persistent failures to translate preclinical findings into clinical success are partially attributed to the limited efficiency of current disease models. Although animal and cellular models have substantially improved our knowledge of the pathological processes involved in these disorders, human brain research has generally been hampered by a lack of satisfactory humanized model systems. This, together with our incomplete knowledge of the multifactorial causes in the majority of these disorders, as well as a thorough understanding of associated (epi)genetic alterations, has been impeding progress in gaining more mechanistic insights from translational studies. Over the last years, however, stem cell technology has been offering an alternative approach to study and treat human brain disorders. Owing to this technology, we are now able to obtain a theoretically inexhaustible source of human neural cells and precursors in vitro that offer a platform for disease modeling and the establishment of therapeutic interventions. In addition to the potential to increase ou...Continue Reading

Citations

Jan 10, 2020·Current Gene Therapy·Christos TsagkarisAmrit Dhar
May 20, 2020·Frontiers in Psychiatry·Yongbo ZhangZheng Zachory Wei
Sep 17, 2021·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Nycole A CoppingJill L Silverman

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