Disease-related structural research
GBA1 binder-design study
GBA1 variants cause Gaucher disease through reduced enzyme activity. This record establishes a public structural baseline for further research.
Public structure analysis · no therapeutic design or experimental validation in this record
Study objective
Generate and computationally evaluate new binder candidates against human GBA1. The existing structural baseline is the first completed phase.
In progress · structural baseline completed · candidate generation not yet started
Research question
Which residues are represented, how is prediction confidence distributed, and which sequence regions are annotated in the source record?
Original predicted structure
Loading public structure…
Existing AlphaFold DB prediction, coloured by pLDDT.
■ ≥90 · ■ 70–90 · ■ 50–70 · ■ <50
Results
Source region annotations
| Signal | 1–39 |
| Chain | 40–536 |
UniProt P04062 · AlphaFold DB AF-P04062-F1 v6
What we calculated
We read one Cα atom per chain and residue from the published PDB file, calculated coverage against the UniProt canonical sequence, and summarised its pLDDT values. The confidence thresholds describe the existing prediction. Full residue-level values and a reproduction script are downloadable.
Interpretation
High structural confidence does not establish enzyme activity, explain an individual variant or demonstrate a treatment. We have not generated a new GBA1 structure or a therapeutic candidate in this record.
Data and reproduction
python3 analyze_gba1.py · Python 3 standard library
Sources and attribution
Structure: AlphaFold DB, EMBL-EBI / Google DeepMind, CC BY 4.0. Sequence and annotation: UniProt, CC BY 4.0. Disease background: U.S. National Library of Medicine, MedlinePlus Genetics.