Learning Path
From curious to contributor. A curated path for anyone who wants to understand HD research and apply AI to it. All resources are free unless noted.
Foundations
Start here. No prerequisites.
What is Huntington's Disease?
HDBuzz explains HD in plain language. Start here before anything technical.
Gene Expression (Khan Academy)
DNA to protein. Central dogma. You need this to understand how the HTT gene causes HD.
Neurobiology of Huntington's Disease
NCBI Bookshelf. The comprehensive reference on HD biology, pathogenesis, and drug discovery approaches.
Genomics + Genetics
Understand the science behind HD at the molecular level.
Introduction to Genomic Technologies
Johns Hopkins. Covers sequencing, alignment, assembly. Foundation for understanding HD genetic research.
Genetics (MIT 7.03)
Genes, chromosomes, genomes, mutation, selection, population genetics, inherited disease. The MIT genetics course.
Genetics and Evolution
Duke. Mutations, DNA repair, population genetics. Directly relevant to understanding CAG repeat expansion.
Python for Genomic Data Science
Johns Hopkins. If you code, this bridges programming to genomics. Directly applicable to our research agents.
Drug Discovery + AI
Where biology meets computation. This is our domain.
Drug Discovery
UCSD. How drugs are found, tested, and approved. Target identification, screening, clinical trials.
Bioinformatics
UCSD. Computational approaches to biology. Sequence analysis, motif finding, genome assembly.
AI for Medicine Specialization
Andrew Ng's team. AI for diagnosis, prognosis, and treatment. Directly connects ML to healthcare.
HD: From Basic Science to Therapeutics
Comprehensive review paper. Covers pathogenesis, biomarkers, therapeutic strategies. Read after completing Level 2.
Contribute
Apply what you've learned. Build something.
Fork HD Research Hub
Clone the repo. Run the agents. Add an experiment. Improve the knowledge base. All code is MIT-licensed.
Join Enroll-HD
The world's largest HD observational study. 30,000+ participants. Your data helps researchers design better trials.
Read the Experiment Reports
See how we applied AI to HD research. Every step documented. Learn by studying real experiments.
Key Concepts to Understand
The HTT gene has a repeated CAG sequence. 36+ repeats cause HD. More repeats = earlier onset. Understanding this is foundational.
CAG repeats grow longer in brain cells over time (somatic expansion). MSH3, FAN1, PMS1 are modifier genes. Hottest target area right now.
The toxic protein produced by the mutated gene. Most therapies aim to lower mHTT levels or clear its aggregates.
ASOs (antisense oligonucleotides) silence the gene. AAV (adeno-associated virus) delivers corrective genes. AMT-130 is an AAV therapy.
Finding new uses for existing FDA-approved drugs. Faster path to patients because safety is already proven. This is what our AI agents do.
A biomarker in blood/CSF that indicates neuronal damage. Used to track HD progression and treatment response.
AI-simulated patients used as a comparison group in clinical trials. Could reduce the need for placebo-only control arms, making trials easier to join.
This site is built by data scientists, not doctors. Nothing here is medical advice. For guidance on HD, please speak with a healthcare provider or visit HDSA.org.