Transcription regulatory elements
AssigneeFreeline Therapeutics Limited
InventorsAzadeh KIA, Romuald CORBAU
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Abstract
The present invention relates to transcription regulatory elements (TREs) such as promoters, which may be used to express a transgene within a cell such as a mammalian cell. The invention further relates to polynucleotides and vectors comprising such transcription regulatory elements, which may be operably linked to a transgene, as well as methods of gene therapy based on using such vectors.
Claims16
A transcription regulatory element comprising a core nucleotide sequence which comprises or consists of a nucleotide sequence having at least 95% identity to SEQ ID NO:
- 2, or a nucleotide sequence which differs from SEQ ID NO:
- 2 by a single nucleotide;
- and wherein:
- a. the transcription regulatory element is between 80 and 280 nucleotides in length and:
- i) does not comprise a nucleotide sequence according to SEQ ID NO:
- 5, or does not comprise at least 20, at least 30 or at least 40 consecutive nucleotides of SEQ ID NO:
- 5;
- and/or
- ii) does not comprise a nucleotide sequence according to SEQ ID NO:
- 4, or does not comprise at least 20, at least 30 or at least 40 consecutive nucleotides of SEQ ID NO:
- 4;
- or
- b. the transcription regulatory element is between 80 and 280 nucleotides in length and further comprises a nucleotide sequence which is located 5′ to the core nucleotide sequence and which has less than 60% identity to a nucleotide sequence comprising at least 20, at least 25, at least 30, at least 35, at least 40 or 45 consecutive nucleotides of SEQ ID NO:
- 4.
The transcription regulatory element as claimed in any one of the preceding claims, wherein the transcription regulatory element comprises a promoter;
- optionally wherein the transcription regulatory element further comprises an enhancer;
- and optionally wherein the promoter is liver-specific.
A polynucleotide comprising a transcription regulatory element as claimed in any one of the preceding claims, wherein the transcription regulatory element is operably linked to a transgene, optionally wherein the transgene encodes a human protein.
Dependencies are inferred from the claim text.