ROCKVILLE, Md., July 31, 2019 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced it entered into a license agreement with Pfizer Inc.
"This license agreement further validates the strength of our intellectual property portfolio and the potential of NAV AAV9 for the treatment of systemic and CNS manifestations of movement disorders," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We are pleased to establish our relationship with Pfizer as they advance this program to develop a potential gene therapy treatment for Friedreich's ataxia."
Thursday, August 1, 2019
Nrf2 Induction Re-establishes a Proper Neuronal Differentiation Program in Friedreich’s Ataxia Neural Stem Cells
La Rosa Piergiorgio, Russo Marta, D’Amico Jessica, Petrillo Sara, Aquilano Katia, Lettieri-Barbato Daniele, Turchi Riccardo, Bertini Enrico S., Piemonte Fiorella; Frontiers in Cellular Neuroscience, vol 3, 2019 DOI=10.3389/fncel.2019.00356
Frataxin deficiency is the pathogenic cause of Friedreich’s Ataxia, an autosomal recessive disease characterized by the increase of oxidative stress and production of free radicals in the cell. Although the onset of the pathology occurs in the second decade of life, cognitive differences and defects in brain structure and functional activation are observed in patients, suggesting developmental defects to take place during fetal neurogenesis. Here we describe impairments in proliferation, stemness potential and differentiation in neural stem cells isolated from the embryonic cortex of the Frataxin Knockin/Knockout mouse, a disease animal model whose slow-evolving phenotype makes it suitable to study pre-symptomatic defects that may manifest before the clinical onset. We demonstrate that enhancing the expression and activity of the antioxidant response master regulator Nrf2 ameliorates the phenotypic defects observed in neural stem cells, re-establishing a proper differentiation program.
Frataxin deficiency is the pathogenic cause of Friedreich’s Ataxia, an autosomal recessive disease characterized by the increase of oxidative stress and production of free radicals in the cell. Although the onset of the pathology occurs in the second decade of life, cognitive differences and defects in brain structure and functional activation are observed in patients, suggesting developmental defects to take place during fetal neurogenesis. Here we describe impairments in proliferation, stemness potential and differentiation in neural stem cells isolated from the embryonic cortex of the Frataxin Knockin/Knockout mouse, a disease animal model whose slow-evolving phenotype makes it suitable to study pre-symptomatic defects that may manifest before the clinical onset. We demonstrate that enhancing the expression and activity of the antioxidant response master regulator Nrf2 ameliorates the phenotypic defects observed in neural stem cells, re-establishing a proper differentiation program.
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