A scalable platform for the development of cell-type-specific viruses
AssigneePresident and Fellows of Harvard College
InventorsSinisa HRVATIN, Mark Aurel NAGY, Michael E. GREENBERG
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Abstract
The technology described herein is directed to adeno-associated vims (AAV) vectors comprising at least one gene regulatory element (GRE) and cells comprising said vectors. In another aspect, described herein are methods of screening for said gene regulatory elements. In another aspect, described herein are nucleic acid compositions comprising a GRE as described herein.
Claims20
An adeno-associated virus (AAV) vector, comprising:
- a. at least one inverted terminal repeat;
- b. at least one gene regulatory element (GRE);
- c. an expression cassette;
- and
- d. a polyadenylation tail.
A method of screening for adeno-associated virus (AAV) cell-type specific gene regulatory elements (GREs), comprising:
- a. labeling a library of GREs with barcodes comprising a nucleic acid, wherein each of the barcodes is associated with a GRE structure, function, or both, in the library of GREs;
- b. packaging the library of labeled GREs into AAV to generate an AAV library;
- c. administering the AAV library to an organism;
- d. detecting the barcodes in one or more cell types in the organism;
- and
- e. identifying the GRE based on the cell type of interest and detected barcodes, thereby screening cell-type specific GREs.
A composition, comprising a nucleic acid sequence at least 70%, 80%, 85%, 90%, 95%, 96%, 97%, 98%, 99% or 100% identical to one of sequence GRE12, GRE19, GRE22, GRE44 or GRE80.
Dependencies are inferred from the claim text.