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Extracellular vesicle-targeted GALT gene therapy in a rat model of classic galactosemia
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Brain GALT restoration by intrathecal injection of scAAV9-hGALT enables partial metabolic correction in adolescent GALT-null rats
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Pharmacological analysis of liver-directed AAV-fVIII gene therapy and anti-fVIII inhibitor incidence in a preclinical model of hemophilia A
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ATF4-Controlled BECN1 Vectors for Treatment of Huntington’s Disease in R6/2 Mice
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Engineering novel chimeric antigen receptors (CARs) for T-cell malignancies using innate immune cells
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Development of a responsive gene therapy to promote clearance of polyglutamine aggregates
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Gene-based neuromodulation for sustained botulinum toxin expression in the treatment of muscle spasticity: a proof of principle study
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