Posted: November 30th, 2015

HEXA hexosaminidase A (alpha polypeptide) [ Homo sapiens (human) ]

Yeast

Tay–Sachs disease.

Mutations in hexosaminidase A, protein.

– non – lethed in utero ( 2-6 years death).

Gene = HEXA.

Advantages = provide correct enzyme for patients. (Current therapy = help to refold           misfolded enzyme.

Problems = getting into brain. – blood/ brain barrier.

Your Strategy = make protein in yeast cells, harvest protein.

– Give to patients – vascular injections, test that going to brain.

– Make human HEXA, to use in “proof of principle’ trails in mice.

– Deliver via blood yeast – made human HEXA to mice.

– Cut up brain + see if protein is in brain cells.