AAV capsid variants for gene therapy
AssigneeUniversity of Florida Research Foundation, Inc.
InventorHugh Lee Sweeney
Life of this patent
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Abstract
Adeno associated viral (AAV) particles are emerging as a useful vehicle for gene delivery to various organs and tissues. Provided here are variant AAV capsid proteins and variant capsid protein containing particles. Compositions of these variant AAV particles can be used to alter tissue tropism and transduction efficiency. In some embodiments, compositions described in this application are useful to produce rAAV particles, and/or to deliver one or more genes of interest to a target tissue.
Claims20
A variant recombinant adeno-associated virus (rAAV) capsid protein comprising the following substitutions:
- (a) A24D, D41N, Q84K, R92K, T158A, K163S, R169K, L189I, A195G, V199L, P201S, S225A, G264S, A269S, R313K, S315N, E350D, Q362E, Q413E, T415S, T417Q, Y447F, T453S, N459G, T462Q, G464L, N471S, T472N, A474S, N475A, T494V, A507G, G508A, N517D, A520V, T528S, D531E, D532G, N540S, N549G, A551G, A555V, D559K, E578Q, 1581Q, T591A, Q594I, I595V, N665S, S667A, N670A, S712N, V722T, and Y733F, wherein the positions of the substitutions are based on the wild-type AAV8 capsid protein amino acid sequence set forth in SEQ ID NO:
- 7;
- or
- (b) K31Q, D41N, G42A, Q84K, R92K, Q105K, T158A, K163S, S180T, P201S, S225A, G264S, A269S, R313K, S315N, E350D, Q362E, Q413E, T415S, T417Q, Y447F, T453S, N459G, T462Q, G464L, N471S, T472N, A474S, N475A, T494V, A507G, G508A, N517D, A520V, T528S, D531E, D532G, N540S, N549G, A551G, A555V, D559K, 1581Q, Q588S, Q589A, Q594I, 1595V, N665S, S667A, N670A, S712N, V722T, and Y733F, wherein the positions of the substitutions are based on the wild-type AAV8 capsid protein amino acid sequence set forth in SEQ ID NO:
- 7.
A variant recombinant adeno-associated virus (rAAV) capsid protein comprising the amino acid sequence set forth in any one of SEQ ID NOS:
- 12, 17, 23, and 29.
A variant recombinant adeno-associated virus (rAAV) capsid protein comprising the amino acid sequence set forth in any one of SEQ ID NOS:
- 13, 19, 25, and 31.
Dependencies are inferred from the claim text.