NewsBIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONSFriday, April 3, 2026 · April 3, 2026
Development and optimization of human glucocerebrosidase-encoding mRNA for Gaucher disease therapy.
WHY IT MATTERS
Recent peer-reviewed research on Gaucher disease that may be relevant for patients and caregivers.
Gaucher disease (GD) is a rare autosomal-recessive lysosomal storage disorder caused by mutations in the GBA1 gene encoding the lysosomal hydrolase glucocerebrosidase (GCase). Mutations in GCase lead to glucosylceramide accumulation within macrophages. Current treatments, including enzyme replacemen...
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