Abstract

Discrepant results due to lack of phenotype-specific species to species relevance to pharmacological results from in vitro cell testing cause inappropriate approaches to treatments to rare conditions in the brain. One such condition is Friedrich Ataxia. The prime focus is on this extreme condition, a neurodegenerative condition that sets precedence for clinical trials. Compounds such as nicotinamide and resveratrol were selected and used in the treatment of Ataxia, the condition shows no improvement. These compounds contracted various responses in iPSC derived neuron generation and more discoverable approach to drug delivery system in vitro conditions. The cell types from iPSC were considered nonrelevant for the disease derived from fibroblasts and more relevant to disease derived from neurons differentiated iPSC cells. This above illustrates the value of utilizing human iPSC cells to improve drug discovery and cure. The over the reacting theory of human iPSC cells are advanced further to discover the potent application of stem cells and further discuss the feasibility of transplantation of Friedrich Ataxia(FA) patients bone marrow-derived mesenchymal stem cells to discover the GAA expansion and reduced FXN and mRNA expression. There is scope for discussion for the assessment of the proliferative ability of pluripotency of FR from bone marrow. The characteristics of bone marrow in vitro differentiation have two types of cells, peripheral neurons, and cardiomyocytes. Also, this chapter leads to series of discussions on the need for testing applications on bone marrow mesenchymal stem cells, mutated gene correction that will play a critical role in gene-based cell therapy, and human artificial chromosomes intact with FXN genes differentiated into neurons or cardiomyocytes.

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