Methods and compositions for regulation of transgene expression
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Publication Number
US-11859190-B2
Publication Date
2024-01-02
Expiration Date
Abstract
Nucleases and methods of using these nucleases for expressing a transgene from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.
Core Innovation
The invention relates to genome editing by providing a non-naturally occurring fusion protein in which a zinc finger DNA-binding protein binds to an endogenous albumin gene and a cleavage domain is coupled to the zinc finger DNA-binding protein. The fusion protein cleaves the endogenous albumin gene. The zinc finger DNA-binding protein comprises 4, 5 or 6 zinc finger domains, and the zinc finger domains include recognition helix regions arranged in a single row in an order corresponding to a table of sequence information.
The disclosed fusion proteins are configured with an albumin gene target, and the cleavage domain enables the fusion protein to cleave the endogenous albumin gene. The document further characterizes the recognition helix region ordering of the zinc finger domains, including sequence identifiers associated with the zinc finger domains and their arrangement.
The related embodiments further include an isolated cell comprising one or more polynucleotides encoding the fusion protein, with embodiments narrowed to stem cells. Additional embodiments include a kit comprising one or more polynucleotides and one or more donor molecules.
Claims Coverage
The independent claim set includes 1 independent claim. The independent claim covers a polynucleotide encoding a zinc finger DNA-binding protein fusion that binds the endogenous albumin gene and cleaves it, with the zinc finger domains restricted to 4, 5 or 6 and arranged with recognition helix regions in a specified table order.
Albumin gene-targeting zinc finger cleavage fusion with ordered zinc finger recognition helix regions
A polynucleotide encoding a non-naturally occurring fusion protein comprising a zinc finger DNA-binding protein that binds to an endogenous albumin gene and a cleavage domain, wherein the fusion protein cleaves the endogenous albumin gene, wherein the zinc finger DNA-binding protein comprises 4, 5 or 6 zinc finger domains, each zinc finger domain comprising a recognition helix region, and wherein the recognition helix regions are in the order shown in a single row in a table.
Overall, the claim coverage focuses on a single core inventive concept: a polynucleotide encoding a non-naturally occurring albumin gene-targeting zinc finger DNA-binding protein fused to a cleavage domain, with zinc finger recognition helix regions arranged in a specified table order for 4, 5 or 6 zinc finger domains. Dependent claims then narrow the context to an isolated cell, further to stem cell categories, and finally to a kit including donor molecules.
Stated Advantages
Documented Applications
No documented applications found
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