摘要:
The present disclosure provides targeting peptides and vectors containing a sequence that encodes targetting peptides that deliver agents, to the eye. The present inventors have discovered peptides that function to target agents, such as viral vectors, to ocular cells. The present disclosure describes a method to utilize these novel peptides to direct, for example, viral capsids to the cell type of interest. In this instance, ocular cells (such as retinal cells) are targeted by the identified peptides. Vectors harboring capsid proteins modified to include such peptides can be used to provide therapeutic agents to the eye.
摘要:
The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus capsids and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.
摘要:
The present invention relates to viral vectors and methods of their production and use. Specifically, the disclosure provides compositions and methods for adeno-associated viral (AAV) vector production, wherein AAV vector comprising chimeric polynucleotides encoding chimeric capsid for improved compositions production. Further disclosed are variable regions (VR) in different AAV serotypes for producing a chimeric capsid, and methods for delivering transgenes using the vectors produced.
摘要:
The invention provides an isolated chimeric virus comprising bocavirus capsid protein and a recombinant adeno-associated viral (AAV) genome, an isolated rBoV comprising human bocavirus capsid protein and a recombinant BoV genome, and uses therefor.
摘要:
The present disclosure provides targeting peptides and vectors containing a sequence that encodes targeting peptides that deliver agents to the brain.
摘要:
The present invention relates to an adeno-associated virus with site-directed mutagenesis and site-specific modification, and a preparation method and uses thereof. Specifically, the present invention uses genetic code expansion techniques to incorporate non-natural amino acid into an adeno-associated virus capsid protein VP1 or fragment thereof, thereby obtaining an adeno-associated virus with site-directed mutagenesis using the non-natural amino acid. The adeno-associated virus with site-directed mutagenesis is equivalent to a wild-type virus in terms of production, transduction and mobility, can couple with other functional molecules, such as targeting molecules, and can carry a functional gene in a normal manner, which indicates that the adeno-associated virus with site-directed mutagenesis can be used as a tool adeno-associated virus, and applied in various aspects associated with adeno-associated virus such as finding adeno-associated virus binding proteins or using as target genetic therapy vector.