Our lead candidate, LYS - GM101, has been designed to replace this defective gene in the cells of GM1 patients, in order to allow for production of the functional
lysosomal acid beta - galactosidase (bgal) enzyme.
Not exact matches
Members of the section use the NIH Clinical Center to evaluate individuals with cystinosis, a
lysosomal membrane defect leading to storage of the amino
acid cystine and causing kidney failure in childhood.
However, no connection has yet been made between the
lysosomal amino
acid transporters and increased mTOR activity in tumour cells.