| abstract |
The present application provides a lentiviral vector used for the treatment of sanfilippo A syndrome, lentivirus, and preparation method and application thereof, wherein the lentiviral vector may be obtained by applying pTYF or modifying a pTYF lentiviral vector at the 5'-end splice donor site and further comprises a SGSH gene. The SGSH gene is specifically connected into the pTYF or the modified lentiviral vector of the present invention, thereby achieving a more efficient gene delivery while ensuring safety, significantly increasing the expression level of the SGSH gene in transgenic brain-related cells, and more efficiently accomplishing the transfer of normal genes during the gene therapy of sanfilippo A syndrome. |