Engineered muscle targeting compositions
AssigneesThe Broad Institute, Inc., President and Fellows of Harvard College, Massachusetts Institute of Technology
InventorsPardis Sabeti, Mohammadsharif Tabebordbar, Simon Ye, Kim Lagerborg, Alexandra Stanton, Amy Wagers
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Abstract
Described herein are muscle-specific targeting moieties and compositions including the muscle specific targeting motifs. Also described herein are uses of the muscle-specific targeting motifs and compositions including the muscle specific targeting moieties. In some embodiments, the muscle-specific targeting moieties and compositions including the muscle specific targeting moieties can be used to direct delivery of a cargo to a muscle cell.
Claims13
A composition comprising:
- a modified AAV capsid polypeptide comprising a targeting moiety capable of conferring a muscle tropism to a capsid or a viral particle
- wherein the targeting moiety comprises one or more n-mer motifs inserted after amino acids 453, 587, and 588 of a wild-type AAV-9 capsid polypeptide, or an equivalent position in a wild-type AAV capsid polypeptide of a different serotype,
- wherein each of the one or more n-mer motifs comprises an amino acid sequence of
- Xm3 Xm2 Xm1 RGD X1 X2 X3 X4,
- wherein Xm3, Xm2, and Xm1 are each independently selected from any one of amino acids A, D, E, G, K, N, R, S, L, and T,
- wherein X1, X2, and X3 are independently selected from any amino acid, and
- wherein X4 is L.
Dependencies are inferred from the claim text.