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The Cranbury, New Jerseybased biotech announced FDA acceptance of its Biologics License Application for RP-L201 (marnetegragene autotemcel), an investigational lentiviral vector (LV)-based gene therapy for LAD-I, a rare, genetic immune disorder that predisposes patients to frequent and often fatal infections

A deficiency of CBS is caused by a mutation of the CBS gene, and results in homocystinuria, a rare inborn error of metabolism in which plasma and urinary homocysteine levels are elevated above normal ranges

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Simvastatin Protects Dopaminergic Neurons Against MPP+-Induced Oxidative Stress and Regulates the Endogenous Anti-Oxidant System Through ERK