I am thrilled to share our latest paper, "AAV8 based gene replacement therapy for hereditary spastic paraplegia type 5," just published in Molecular Therapy - Methods & Clinical Development!
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Warm wishes for a wonderful holiday season and a New Year that’s full of creativity, inspiration, and achievement.
Help shape the future of neurodegeneration research – in a leading role at DZNE and the University Tübingen!
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dlvr.it
AAV8-mediated gene therapy targeting the liver effectively normalized peripheral oxysterol levels in a mouse model of hereditary spastic paraplegia type 5 (SPG5). However, brain oxysterol levels remained partially corrected, indicating that successful SPG5 treatment requires a gene therapy approach that also targets the central nervous system.