Intellectual Merit. The totipotent state exists during a brief period of embryonic development soon after fertilization before implantation. In contrast to pluripotent embryonic stem cells (ESCs) or reprogrammed iPSCs, which have the potential to generate all embryonic tissue types, totipotent cells can produce both embryonic and extraembryonic tissues. To date, the totipotent state can be achieved by natural fertilization, or artificially by somatic cell nuclear transfer (aka cloning) or removal of epigenetic regulators in iPSCs.

Genetic testing has greatly advanced diagnosis of early onset epilepsy. In spite of the expanding number of monogenetic disorders, such as epilepsy in patients with Aristaless-related homeobox gene (ARX) mutations, many of these rare diseases do not have a validated experimental model and thus suffer from limited understanding of the mechanisms of brain development and epileptogenesis. ARX is a transcription factor which is widely expressed in the developing and adult brain, skeletal muscle, pancreas, and testes.

OVERVIEW

This contract evaluate biological membrane composition and functional protein presence and activity with the goal of developing products for tissue regeneration.

Our findings will help clarify the signaling pathways associated with the developmental changes and activation of aging processes occurring during muscle over an extended time in vitro. Given this, we will be able to determine the optimal time window when these precursors have the highest potential to efficiently engraft a host muscle. Txbiomed Project: 4673

The work proposed in this application is a pilot study. The data acquired will be used to build the rationale for a larger application (RO1) to the NIH-NEI. Overall, this study explores the use of a large non-human primate, specifically Baboon pluripotent stem cells to a derive RPC system for translational stem cell therapy investigations. The results obtained will allow for experimental treatment development for naturally occurring non-human primate (Baboon) retina degeneration identified in the local Baboon population. This work will be directly translational to humans.

In this application we propose one pilot study to test the efficacy of materials provided by Dr. Ramon Coronado from LSMRI to prevent disease progression in an experimental model of multiple sclerosis (MS) in mice in Dr. Forsthubers laboratory at UTSA.