Enzyme replacement therapy for lysosomal storage diseases
Project ID : 10-2013-555
Lysosomal Storage diseases (LSD) are inherited genetic defects, resulting in an enzyme deficiency.
This deficiency prevents the lysosome from metabolizing cellular waste, and results in their accumulation in the cell. Excessive storage of proteins, saccharides and/or fats can cause permanent cellular and tissue damage, particularly in the brain, peripheral nervous system, liver, spleen, and bone marrow.
Additional information can be provided upon request.
Project manager
Adi Elkeles
BD Manager
Project researchers
Ruth, Emeritus Navon
T.A.U Tel Aviv University, Medicine-Sackler Faculty
Human Molecular Genetics
Naftali Stern
Sourasky Medical center,
Anthony Futerman
Weizmann Institute of Science (WIS),
Ari Zimran
,
Etty Osher
Sourasky Medical center,
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