The invention discloses an overexpression vector of LAMP2B and application thereof, which can effectively
delay the
disease progress of DMD, and comprises: (a) an AAV vector which is an adeno-associated
virus vector and has high
muscle tissue targeting and low liver
tropism; (b) a CMV (cytomegalovirus)
promoter, wherein the CMV
promoter is a cytomegalovirus
promoter; (c) a coding sequence of an hLAMP2B
gene, namely a coding sequence of a human LAMP2B
gene, and the coding sequence of the hLAMP2B
gene is operably linked to the CMV promoter; wherein the vector is configured to overexpress LAMP2B
protein in muscular tissue, and the vector is self-complementary AAV, namely scAAV, the concept is ingenious, the novel AAV vector with high
muscle transformation efficiency is utilized to deliver a complete LAMP2B gene (LAMP2B is a gene related to
autophagy regulation) into
muscle cells of all organs of a mouse (AAV-hLAMP2B for short), and the
autophagy regulation function of the mouse is improved. The
muscle tissue function of the mouse DMD
disease model is comprehensively assisted to recover, and the
disease progress of the DMD is delayed.