Non-naturally occurring capsids for delivery of nucleic acids and/or proteins
AssigneesThe Broad Institute, Inc., Massachusetts Institute of Technology
InventorsFeng Zhang, Serge Gueroussov
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Abstract
Provided herein are non-naturally occurring self-assembling polypeptides for transferring nucleic acids and/or proteins to a cell, pharmaceutical compositions comprising such polypeptides, and methods for treatment comprising use of such compositions. The methods for producing polypeptide compositions may include combining in a solution, unassembled recombinant GAG-like proteins, nucleic acids and/or proteins in low salt conditions; and increasing the ionic strength of the solution.
Claims30
A engineered delivery system comprising:
- a. a capsid comprising one or more Gag-homology proteins or functional domains thereof;
- and
- b. a therapeutic cargo heterologous to the capsid captured by, or packaged within, the capsid.
The delivery system of claim 3, wherein the export compartment domain is engineered to provide cell-specific uptake or delivery efficiency.
A method of preparing a delivery system comprising:
- incubating one or more Gag-homology proteins or functional domains thereof with a heterologous cargo in conditions suitable for capsid formation, thereby packaging the cargo in a capsid comprising the Gag polypeptide.
A method of delivering a heterologous cargo to a human cell comprising administering a delivery system comprising the heterologous cargo packaged within a capsid comprising one or more Gag-homology proteins or functional domains thereof.
Dependencies are inferred from the claim text.