Self-assembling systems for modeling intracellular protein aggregation
OwnerViviana Gradinaru, Yujie Faninventors
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Abstract
Disclosed herein include polynucleotides, expression vectors and compositions enabling expression of a self-assembling proteopathic fusion protein comprising a self-assembling domain fused to a proteopathic polypeptide associated with a proteinopathy. The self-assembling proteopathic fusion protein is capable of forming intracellular protein aggregates that enable robust recapitulation of disease pathology. Provided herein also include methods of using the self-assembling systems disclosed herein to induce proteinopathies (e.g., neurodegenerative diseases) and to screen therapeutic compounds for the treatment of proteinopathies.
Claims26
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An expression vector, comprising:
- one or more first promoters operably connected to a first polynucleotide comprising a first nucleic acid unit encoding a proteopathic polypeptide and a second nucleic acid unit encoding a self-assembling domain,
- wherein the first polynucleotide, upon expression in a cell, encodes a self-assembling proteopathic fusion protein comprising the proteopathic polypeptide connected to the self-assembling domain and capable of forming intracellular protein aggregates in the cell.
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An expression system, comprising:
- a first expression vector comprising a first promoter operably connected to a first polynucleotide comprising a first nucleic acid unit encoding a proteopathic polypeptide and a second nucleic acid unit encoding a self-assembling domain;
- and
- a second expression vector comprising a second promoter operably connected to a second polynucleotide encoding a transactivator,
- wherein the first promoter is an inducible promoter capable of inducing transcription of the first polynucleotide in the presence of the transactivator and a transactivator-binding compound, and
- wherein the first polynucleotide, upon expression in a cell, encodes a self-assembling proteopathic fusion protein comprising the proteopathic polypeptide connected to the self-assembling domain and capable of forming intracellular protein aggregates in the cell.
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Dependencies are inferred from the claim text.