Abstract: A nucleic acid construct for treating hereditary coagulation factor deficiency and a novel use thereof are provided. The construct includes the following elements: a polynucleotide encoding coagulation factor IX; and an AAV cis-regulatory element. An adeno-associated virus prepared from the nucleic acid construct and a nucleic acid construct carrying a coagulation factor target gene may have a muscle targeting property in an intramuscular injection delivery mode, and help express the coagulation factor at a therapeutically effective amount.

Abstract: Provided herein are a nucleic acid construct encoding an anti-VEGF fusion protein, such as an expression vector, as well as recombinant virus such as a recombinant lentivirus and a recombinant adeno-associated virus prepared by the construct. Also provided herein are uses of the fusion protein, the nucleic acid construct, and the recombinant virus in the treatment of age-related macular degeneration (AMD), such as wet AMD.

Abstract: Disclosed herein are gene therapy vectors used for efficiently transducing cells to express a human ?-globin gene. Specifically disclosed is an expression vector which comprises: an expression cassette for a ?-globin gene, which comprises exons and introns of human ?-globin gene, as well as cis-acting elements including one or more of WPRE, SV40 polyadenylation signal and/or SV40 ori. The disclosed expression vectors have significantly enhanced viral vector packaging efficiency in viral vector packaging cell lines, which leads to effective integration of lentiviral vectors and high expression level of ?-globin gene in target cells. Also disclosed are pharmaceutical compositions and therapeutic methods utilizing such expression vectors.

Abstract: A nucleic acid construct for improving an adeno-associated virus yield, and a construction method therefor. The nucleic acid construct includes: an adeno-associated virus (AAV) element, and a polynucleotide encoding an IE protein. Said AAV element includes a polynucleotide encoding a Cap protein, a polynucleotide encoding a Rep protein, and an AAV cis-regulatory element. The construction method includes integrating an AAV element carrying an exogenous target gene and a polynucleotide that encodes the IE protein into to a baculovirus vector backbone. The obtained recombinant adeno-associated virus (rAAV) has a low empty capsid rate, while the rAAV yield of a single cell and a unit volume culture is increased, the production cost is reduced, and the production is easy to scale up.