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!
@dzne.science @hih-tuebingen.bsky.social @tueneurocampus.bsky.social
Stefan Hauser
#MTMCD
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.
