Regulatory nucleic acid sequences
AssigneeAsklepios Biopharmaceutical, Inc.
InventorsTony Oosterveen, Anne Braae, Sinclair Cooper, Juan Manuel Iglesias, Michael L. Roberts
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Abstract
The present invention relates to regulatory nucleic acid sequences, in particular CNS—specific promoters, and elements thereof. The invention also relates to expression constructs, vectors, virions, pharmaceutical compositions and cells comprising such promoters and to methods of their use. The regulatory nucleic acid sequences are of particular utility for gene therapy applications.
Claims34
A synthetic CNS-specific promoter comprising a sequence according to any one of SEQ ID NOs 1-8, 21-26 or a functional variant thereof.
A CNS-specific cis-regulatory element (CRE) comprising a sequence according to any one of SEQ ID NOs:
- 9-11, 28-31, or a functional variant thereof.
An isolated minimal or proximal promoter comprising a sequence according to any one of SEQ ID NOs:
- 12-13, or a functional variant thereof.
Dependencies are inferred from the claim text.