The research efforts at the Center for Rare Neurological Diseases (CRND) are based on the premise that by exploring the molecular basis of rare diseases, new molecular targets can be identified for assessment in common diseases.
CRND will focus on developing targeted treatments for neurodegenerative diseases that affect adults and children, including Alzheimer's disease (AD), Parkinson’s disease (PD), Amyotrophic Lateral Sclerosis (ALS), Huntington’s disease (HD), and rare lysosomal disorders.
Clinical research in neurodegeneration is for the most part completely dependent on preclinical studies to identify pathways and targets for therapeutic development. Our recent discoveries have provided a platform for new approaches to therapeutic development for both rare and common neurodegenerative diseases.
By focusing on rare lysosomal diseases that are clinically and genetically linked to more common neurodegenerative conditions, the Center has an unprecedented opportunity to identify novel therapeutic targets for these disorders. In pursuing the development of targeted therapies for Parkinson’s disease, Alzheimer’s disease, and other dementias, the CRND looks to position Northwestern Medicine a leader in development of personalized treatments for neurodegenerative diseases.
Areas of Research
The main goal of CRND is to explore clinical and genetic links between rare and common disorders in order to uncover specific targets and pathways for therapeutic development. We have utilized methodological advances, including the generation of human neurons from patient-specific skin fibroblasts, which together with gene correction strategies offer the most relevant model system for studies of disease mechanisms and development of new therapeutics for neurodegenerative disorders.
Find more information about our areas of focus via the links below.
- Therapeutic Strategies
Learn more about the types of therapies we intend to pursue
- Induced Pluripotent Stem Cell and Human Neurons
Discover our current abilities and long-term goals related to studies in human neurons
- Drug Development and Delivery
See our approach to overcoming therapy delivery challenges
Find our faculty's recent noteworthy publications on rare neurologic diseases
Contact us for more information about the Center for Rare Neurological Disorders’ research or opportunities for collaboration.