Gaucher disease: who works on it

One card per institution. Pick a target or a country to narrow the list.

• All targets
• GBA1 5
• LRRK2 1
• MEK 0
• NRF2 0
• SNCA 0

5 of 5 institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Funded research projects that name this disease in their title, terms or summary and have not ended yet: who is funded, where, how much and until when. Organisations only; the funder's page has the details.

• NIH NIGMS · R35

  Simultaneous Separation, Identification, and Quantification of Lipid Isomers using Non-Covalent Chemistry-Based Enhancement of Tandem Ion Mobility-Mass Spectrometry

  Brigham Young University· Provo, United States· US$406,576 awarded in fiscal year 2026· until May 2031

• NIH NICHD · K08

  Bioengineered extracellular vesicles as a therapeutic platform for inborn errors of metabolism

  Brigham and Women's Hospital· Boston, United States· US$171,720 awarded in fiscal year 2026· until Aug 2031

• NIH NIDDK · R43

  Extended In Situ Production of Glucocerebrosidase (GCase) for the Treatment of Gaucher Disease

  Keylicon Biosciences Inc· Brookline, United States· US$400,000 awarded in fiscal year 2026· until Aug 2027

• NIH NINDS · R21

  Identification of the Palmitoyltransferase for Glucocerebrosidase Trafficking Receptor LIMP2 and Restoration of GBA1 Function in Parkinson Disease and Lewy Body Dementia.

  Brigham and Women's Hospital· Boston, United States· US$492,250 awarded in fiscal year 2026· until Jul 2028

• NIH NCATS · R43

  Engineered Tregs as a therapy to Gaucher syndrome

  Celdara Medical, Llc· Lebanon, United States· US$310,564 awarded in fiscal year 2026· until Jun 2027

• European Commission (Horizon Europe) · HORIZON-RIA

  Reconstruction and Computational Modelling for Inherited Metabolic Diseases (Recon4IMD)

  University of Galway· Galway, Ireland· EU contribution €7,871,903· until May 2027· 13 countries

  The disease is named only in the funder's project summary, not in the title.

6 of 23 running projects, newest first. Listed amounts together: US$12.5m + €7.9m.

Sources: NIH RePORTER (U.S. National Institutes of Health, public domain) and CORDIS (European Commission, CC BY 4.0). Changes: filtered by disease, organisations matched to ROR.

Trials whose registry record says individual participant data (IPD) will be shared.

• Parkinson's Disease in Type 1 Gaucher Disease

  NCT07886879· Icahn School of Medicine at Mount Sinai· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Organoids 2
• CRISPR-Cas Systems 1
• Proteomics 1
• Metabolomics 1
• Magnetic Resonance Imaging 3
• Cohort Studies 1
• Genome-Wide Association Study 2

• A genome-wide screen identifies that PLCG2 restrains lysosomal GCase activity.

  Proc Natl Acad Sci U S A, Jul 2026

• Genomic Structural Equation Modeling Provides an Initial View of the Genetic Architecture Related to Type 1 Gaucher Disease.

  Hum Mutat, Apr 2026

See which targets of this disease your abstract mentions, and go to the people working on them.