Integrated drug discovery platform for inborn error of metabolism disorders
OwnerEhud GAZIT, Dana LAORinventors
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Abstract
The invention relates to in vivo systems and high throughput screening platform for drugs applicable in inborn error of metabolism (IEM) disorders. More specifically, the invention provides yeast screening system of candidate therapeutic compounds applicable in IEM disorders associated with accumulation of at least one metabolite. The systems of the invention comprise yeast cell/s that carry at least one manipulation in at least one yeast metabolic pathway, that leads to accumulation of said metabolite.
Claims20
A yeast screening system for candidate therapeutic compounds, said system comprises:
- (a) a yeast cell and/or yeast cell line, and/or yeast cell population, that carry at least one manipulation in at least one yeast metabolic pathway, that leads to accumulation of at least one metabolite, wherein accumulation of said at least one metabolite is associated with at least one inborn error of metabolism (IEM) disorder;
- and optionally
- (b) at least one reagent or means for determining at least one of, the accumulation of said metabolite and at least one phenotype associated with accumulation of said metabolite.
A screening method of candidate therapeutic compounds for treating, preventing, ameliorating, reducing or delaying the onset of at least one IEM disorder associated with accumulation of at least one metabolite, the method comprising the steps of:
- (a) contacting a manipulated yeast cell and/or yeast cell line, and/or yeast cell population, with a candidate compound, said yeast cell and/or yeast cell line, and/or yeast cell population, carry at least one manipulation in at least one yeast metabolic pathway, that leads to accumulation of said metabolite;
- (b) determining in the contacted cells of (a), at least one of, the accumulation of said metabolite and the level of at least one phenotype associated with the accumulation of said metabolite;
- and
- (c) determining that said candidate is a therapeutic compound for said IEM disorder if at least one of, the accumulation of said metabolite and said phenotype is modulated as compared with the accumulation of said metabolite and phenotype in the absence of said candidate compound.
Dependencies are inferred from the claim text.