Available tools (2026)

44 genotypes total, comprising 13 patient-derived genotypes (GBA1, LRRK2, TMEM175), 20 CRISPR knock-ins (GBA1 p.E326K, p.N370S,
p.L444P, rs3115534; TMEM175 modifier (p.M393T); SNCA p.A53T; heterozygous and homozygous, male and female), and 11 CRISPR knock-outs (GBA1, GBA1LP, LRRK2, TMEM175, SNCA, MAPT, ASAH1, GALC, CTSB, SMPD1, SCARB2)

In development

Additional rare variants (p.T408M in production); compound heterozygotes; expanded sex-balanced knock-in panels

Access through

https://cbigr-open.loris.ca/
https://hpscreg.eu/browse/provider/1355

Zebrafish

Available tools (2026): gba1 p.W378G knock-in (validated, stable germline transmission confirmed); gba1 KO (frameshift)

In development: Expansion to WT/Het/Homo cohorts; p.D409S variant line (analogous to human p.N409S); additional gba1 variants

Access through: Zebrafish Core (MNI)

C. elegans

Available tools (2026): gba-3 deletion mutants; gba-3 transgenic ubiquitous overexpression strain; gba-3 transgenic pan-neuronal overexpression strain; worms ubiquitously expressing human wild-type GBA1, human GBA1 (p.L444P), and human GBA1 (p.N370S); validated GCase assays

In development: PD models (SNCA, LRRK2, etc.) with increased or decreased GCase activity

Access through: G-Can via MTA

Mouse

In development: TMEM175 p.I390T knock-in; inducible TMEM175 knockdown model (in collaboration with MJFF)

Access through: To be confirmed

67 antibodies characterized across 6 targets (GCase, GALC, SCARB2, SMPD1, PSAP, TMEM175); datasets openly available

https://zenodo.org/communities/ycharos/

Available tools (2026)

GBA1 patients, relatives, controls across multiple cohorts including C-OPN, QPN, PD GENEration, and international partners.

In development

Healthy carrier recruitment to 10,000

Access through

NeuroHub, C-OPN, QPN, Genome Canada

Links:

https://pdgenetics.shinyapps.io/GBA1browser/
https://b4ynee.shinyapps.io/GBA1PDviewer/