Lentiviral vectors for therapeutic expression of btk in haematopoietic cells
AssigneeNoga Therapeutics Ltd.
InventorsLiron ELKOUBY, Noam DIAMANT, Liat SHACHNAI-PINKAS
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Abstract
The present application, in some embodiments, is directed to a polynucleotide including: (a) a first nucleic acid molecule including a sequence of a human endogenous Bruton's tyrosine kinase (BTK) promoter; and (b) a second nucleic acid molecule including a codon optimized sequence encoding a BTK or a functional analog thereof. Further provided are an expression vector, a cell, and a composition, all of which include the polynucleotide of the invention, and a method of using same, such as for treating X-linked Agammaglobulinemia (XLA) in a subject in need thereof.
Claims20
A polynucleotide comprising:
- a. a first nucleic acid molecule comprising a sequence of between 799 to 1,533 nucleotides of a human endogenous Bruton's tyrosine kinase (BTK) promoter;
- and
- b. a second nucleic acid molecule comprising a codon optimized sequence encoding a BTK or a functional analog thereof,
- wherein said first nucleic acid molecule and said second nucleic acid molecule are operably linked, and wherein said codon optimized is for BTK expression in a subject, cell derived therefrom, or both.
Dependencies are inferred from the claim text.