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US 11,920,150 B2Granted
Engineered muscle targeting compositions
AssigneesThe Broad Institute, Inc., President and Fellows of Harvard College, Massachusetts Institute of Technology
InventorsPardis Sabeti, Mohammadsharif Tabebordbar, Simon Ye
Life of this patent
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Abstract
Described herein are targeting moieties that can be capable of specifically targeting muscle cells and can include an n-mer motif. In some embodiments, the n-mer motif contains an RGD motif. Also described herein are vector systems, particles, polypeptides that can encode and/or contain one or more targeting moieties. Also described herein are methods of delivering a cargo to a cell, such as a muscle cell, using one or more of the targeting moieties described herein.
Claims30
A composition comprising:
- a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, wherein the n-mer motif is an RGD motif, wherein the RGD motif has a formula of XmRGDXn, wherein each instance of X is independently selected from any amino acid, m is 0-4 amino acids and n is 1-15 amino acids or m is 1-4 amino acids and n is 0-15 amino acids, wherein the targeting moiety comprises a viral capsid protein, wherein the n-mer motif is (a) inserted between any two contiguous amino acids of the viral capsid protein, (b) replaces one or more native viral capsid protein amino acids, or both (a) and (b);
- and
- a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
Dependencies are inferred from the claim text.