Novel therapeutic approaches: Rett syndrome and human induced pluripotent stem cell technology

• Published in: Stem cell investigation Vol. 4; no. 3; p. 20
• Main Authors: Gomathi, Mohan, Balachandar, Vellingiri
• Format: Journal Article
• Language: English
• Published: China AME Publishing Company 2017
• Subjects: Review; Indian Traditional Ayurvedic System; ayurvedic medicines; drug discovery; neurodevelopmental disorder; Human induced pluripotent stem cells (hiPSC); active compounds; Rett syndrome (RTT)
• ISSN: 2306-9759; 2313-0792; 2313-0792
• DOI: 10.21037/sci.2017.02.11

Recent advances in induced pluripotent stem cell (iPSC) technology target screening and discovering of therapeutic agents for the possible cure of human diseases. Human induced pluripotent stem cells (hiPSC) are the right kind of platform for testing potency of specific active compounds. Ayurveda, the Indian traditional system of medicine developed between 2,500 and 500 BC, is a science involving the intelligent formulations of herbs and minerals. It can serve as a "goldmine" for novel neuroprotective agents used for centuries to treat neurological disorders. This review discusses limitations in screening drugs for neurological disorders and the advantages offered by hiPSC integrated with Indian traditional system of medicine. We begin by describing the current state of hiPSC technology in research on Rett syndrome (RTT) followed by the current controversies in RTT research combined with the emergence of patient-specific hiPSC that indicate an urgent need for researchers to understand the etiology and drug mechanism. We conclude by offering recommendations to reinforce the screening of active compounds present in the ayurvedic medicines using the human induced pluripotent neural model system for research involving drug discovery for RTT. This integrative approach will fill the current knowledge gap in the traditional medicines and drug discovery.