Axonome
Neurodegeneration Explained
Axonome comes from two words: axon and tome. An axon is the long arm of a neuron that carries signals to other cells, allowing the nervous system to communicate. A tome is a substantial book or body of knowledge. Together, Axonome reflects the goal of this site: to serve as a clear, organized, and scientifically grounded resource on neurodegenerative disease. It is designed for patients, families, students, trainees, clinicians, and researchers by making the core ideas understandable without a scientific background while still providing enough depth for readers with medical or scientific training.
This project was motivated by my grandfather’s struggle with neurodegenerative disease and by a desire to make the science behind these conditions more accessible, organized, and useful.
Explore the cellular drivers of neurodegeneration including protein aggregation, mitochondrial stress, and inflammation.
See the cutting edge of disease understanding, drug discovery, and clinical therapies.
Diseases
Explore the presentation, progression, and pathology of common neurodegenerative diseases.
Investigate the most common neurodegenerative disease worldwide, including the role of amyloid plaques and the immune response.
Learn about the devastating monogenic neurodegenerative disease defined by proteing aggregation.
Understand the common movement disorder caused by alpha-synuclein aggregation and loss of dopamine.
Understand the role of neuroinflammation, demyelination, and remyelination pathways.
Explore the spectrum of motor neuron disease and frontotemporal dementia and their shared causes.
Mapping the genetic architecture and specific risk variants associated with neurodegenerative onset.
Clinical Trial Updates & Research Summaries
Clinical Trial Updates
AMT-130 is a signal dose therapy that requires surgery to deliver. It is an adeno-associated virus (AAV), which are effective at delivering genes without causing disease or infection. This AAV encodes a micro RNA that lowers the levels of huntingtin protien... read more
Research Summaries
Foldamers are synthetic molecules that spontaneously fold into specific shapes when in solution. Here, the authors used a foldamer called SK-129 to bind to alpha-synuclein aggregates. In Parkinson’s models of both worms and cultured human neurons, treatment with SK-129 improved many hallmarks of disease including number of dopaminergic neurons and ROS levels... read more