AAV gene-replacement therapy for DYT-SGCE myoclonus-dystonia.
- AAV
- AlphaFold
- Structural bio

Patient-specific molecular identity should drive therapy design, not population averages.
The concrete test case is a single frameshift in the SGCE gene1 — one nucleotide duplicated, one premature stop codon, one truncated ε-sarcoglycan. Arcivus Lab's first program is an AAV vector that replaces the broken copy. No small molecules. No symptom management.
NorthProt is the silicon half: Python, Claude API, MCP pipelines, pgvector retrieval over the primary literature. Arcivus is the carbon half: capsid engineering, construct design, wet-lab validation. The same person runs both sides because the target-identification-to-construct gap2 is where rare-disease programs die, and collapsing it is a tooling problem before it is a biology problem.
From first principles, source open, no ceremony3 — this is how the work is done, not how the site is marketed. Every tool Arcivus builds ships open-source. Every decision is traceable to a paper or a sequence. Small. Real.
AAV gene-replacement therapy for DYT-SGCE myoclonus-dystonia.
Solo R&D lab. Carbon and Silicon. 100% upstream of Arcivus.
Open-source 3D visualization of the SGCE protein for understanding the DYT-SGCE c.108dup mutation.
Open-source MCP server for Brightspace LMS — automated coursework retrieval via Playwright.
Jan 12, 2025
Myoclonus-dystonia is a SGCE-driven movement disorder with reduced striatal D2 receptor density and excessive dopamine release. Restoring D2 signaling — pharmacologically, genetically, or via lifestyle — is a plausible therapeutic axis, and the review surveys the evidence for each.
This page was designed and implemented end-to-end by Claude (Opus 4.6). Sourcing, copy decisions, layout, and code generation were all model-driven from a single brief. The human pipeline — review, deploy, infrastructure — is by Cyan.
Set in Source Serif 4 and JetBrains Mono. Cream paper. Cool ink. ε-sarcoglycan ribbon from AlphaFold prediction AF-O43556; 3Dmol.js on desktop.
Source on GitHub© 2026 NorthProt