Main > PROTEINS > Proteomics > Human Proteomics > FKBP12 Protein > Mutant Form > Therapeutic Protein Deliv

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TECHNOLOGY The method [called "RAPID" (See Abbreviation)] is based on a mutated form of a soluble human protein called FKBP12 The mutant forms dimers that completely dissociate on addition of a synthetic ligand that can be given orally. The researchers engineered a gene to produce proteins contg many copies of the dimer-forming region of their mutant protein joined by an enzyme-cleavable linkage to a therapeutic protein
When the engineered gene was injected into cells, the resulting protein clumped together to form large aggregates in the cells' endoplasmic reticulum, the first compartment of the normal pathway for secreting proteins. Addition of the synthetic ligand dissolved the aggregates, & the released proteins were secreted from the cells in the normal way. As part of that process, cellular enzymes cleaved the therapeutic portion from the rest of the protein so that it was secreted from the cell in its active form.
It's a combination of gene therapy & chemistry that allows chemical biology to be used in a therapeutic way
UPDATE 02.00
USES - GH delivery
- Insulin delivery
- Pain-relief endorphin deliv
- Etc
AUTHOR This data is not available for free
LITERATURE REF. This data is not available for free

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