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US 2024/0200095 A1Published application
Promoters
AssigneeFreeline Therapeutics Limited
InventorsAzadeh KIA, Samantha CORREIA
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Abstract
The present invention relates to transcription regulatory elements (TREs) based on the combination of a hAAT sequence element and an AMBP sequence element and which promote greater expression than HCR-hAAT. The invention further relates to compositions comprising the AAV vectors, as well as methods of gene therapy based on the use of such vectors and compositions.
Claims26
An adeno-associated virus (AAV) vector comprising a vector genome comprising a transcription regulatory element (TRE) that:
- (a) comprises a human alpha-1-antitrypsin (hAAT) sequence element;
- (b) comprises a human alpha-1-microglobulin/bikunin precursor (AMBP) sequence element;
- and
- (c) promotes greater expression than hepatic control region-hAAT (HCR-hAAT), wherein the vector genome is between 2.2 and 5.0 kbp in length.
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Dependencies are inferred from the claim text.