Ardat

Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47

“We are pleased to report the publication of our recent paper at EMBO Molecular Medicine. The work describes the progress made in gene therapy approach for spastic paraplegia type 47 (SPG47): from discovery to IND-enabling studies including safety and toxicity investigations. Further details about the publications can be accessed via the link: Read the publication here.
Article accepted 19/09/2024