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• W2894050590 abstract "OBJECTIVE: In vivo comparison of the efficacy of human stem cells derived neuronal cells in non-human primate and rodent models of Parkinson9s disease. BACKGROUND: Human parthenogenetic stem cells (hpSCs) are pluripotent stem cells that have an important clinical advantage when used in cell replacement therapy because of their homozygosity at the HLA loci, which simplifies immune matching with potential significantly reduced immunogenicity. As such, hpSC are useful for generation of dopaminergic neurons for Parkinson9s disease cell therapy. DESIGN/METHODS: Highly pure populations of neural stem cells (NSCs) and dopaminergic (DA) neurons were generated from hpSCs and transplanted into rodent and non-human primate models of Parkinson9s disease. In the rodent model, behavioral improvement is assessed in 6-OHDA rats with the cylinder test, amphetamine and apomorphine induced rotation tests. In the non-human primate model, behavioral improvement is assessed in MPTP-induced African green monkeys with parkinsonian scores. The intake of food and water is monitored on a daily basis and body weight is recorded weekly. Cell engraftment, viability and phenotype of the implanted cells are determined through histology and HPLC at the end of the studies. Tumorigenicity and safety of the therapy is assessed at the end of both studies by gross necropsy. RESULTS: Preliminary results of the animal studies have shown that the neuronal cells engraft in a Parkinsonian rat model, release dopamine in vivo , and do not give rise to tumors in the brain. We are currently evaluating the post-implantation behavioral improvement of transplanted NSCs and DA neurons precursors in rodent and non-human primate models of Parkinson9s disease. CONCLUSIONS: The results of our pre-clinical animal studies indicate that neuronal cells derived from human parthenogenetic stem cell can be a viable alternative for the treatment of Parkinson9s disease. Supported by: International Stem Cell Corporation. Disclosure: Dr. Semechkin received personal compensation as an employee of International Stem Cell Corporation. Dr. Semechkin has received personal compensation for serving as a Director of International Stem Cell Corporation. Dr. Semechkin holds stock and/or stock options in International Stem Cell Corporation, which sponsored reseaerch in which Dr. Semechkin was involved as an investigator. Dr. Garitaonandia has received personal compensation for activities with International Stem Cell Corporation as an employee. Dr. Gonzalez has received personal compensation for activities with International Stem Cell Corporation as an employee. Dr. Ostrowska has received personal compensation for activities with International Stem Cell Corporation. Ms. Abramihina has received personal compensation for activities with International Stem Cell Corporation as an employee. Dr. Wambua has received personal compensation for activities with International Stem Cell Corporation as an employee. Dr. Noskov has received personal compensation for activities with International Stem Cell Corporation as an employee. Dr. Boscolo has nothing to disclose. Dr. Laurent has nothing to disclose. Dr. Sanchez-Pernaute has received personal compensation for activities with International Stem Cell Corporation as a consultant. Dr. Snyder has received personal compensation in an editorial capacity as Section Editor, Experimental Neurology, Editor, Current Protocols in Stem Cell Biology." @default.
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• W2894050590 date "2013-02-12" @default.
• W2894050590 modified "2023-09-27" @default.
• W2894050590 title "In Vivo Efficacy Study of Stem Cell Derived Neuronal Cells for the Treatment of Parkinson's Disease (S23.007)" @default.
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