Degenerative Diseases

Brain enzyme is double whammy for Alzheimer’s disease

Brain enzyme is double whammy for Alzheimer’s disease

Dr. Huaxi Xu's research discovered that BACE1, an enzyme that produces b-amyloid also regulates another cellular process that contributes to memory loss.

Searching for causes of neuron death in Alzheimer’s and TBI

Searching for causes of neuron death in Alzheimer’s and TBI

Sanford-Burnham researchers discovered that the protein appoptosin prompts neurons to commit suicide in several neurological conditions.

Using stem cells to treat Parkinson’s disease

Using stem cells to treat Parkinson’s disease

Dr. Stuart Lipton has discovered a possible method to use MEF2C-programmed human stem cells for treatment of Parkinson's disease.

What are neurodegenerative diseases?

Despite significant progress in decoding the human genome and identifying the genetic basis of many monogenic diseases, there is still a tremendous void in understanding the basis of age-associated complex diseases, two of the most common being Alzheimer’s disease and Parkinson’s disease. It appears that inappropriate 3-dimensional protein folding is a hallmark of these diseases and deteriorates with environmental changes and age. More importantly, the cell processes that survey the quality of protein folding and damage, and those that regulate degradation also deteriorate with age and are associated with degenerative diseases.

What is the focus of our program—What questions are we asking?

Our research is focused to understand how the cell discriminates functional from nonfunctional proteins to target the latter for degradation. The answers to these questions will lead to novel therapeutics for many diseases associated with aging. We study intrinsic cellular mechanisms that recognize misfolded/damaged proteins and target their degradation. Importantly, our findings have demonstrated the damaging impact of oxidative/nitrositative stress on protein structure and function in disease pathogenesis, for example the neurodegenerative diseases of Alzheimer’s and Parkinson’s, and their impact on neural synaptic structure and signaling. Finally, defective protein folding and/or degradation are now implicated in many diseases ranging from cancer to metabolic and inflammatory response syndromes.

How will our research help patients?

Scientists in this program paved the way for development of a drug for Alzheimer’s disease that, for the first time, prevents brain cell death rather than merely asking symptoms caused by the loss of these cells. A number of other strategies are now being developed that target protein folding/degradation pathways for preserving cell function in the brain and other vital organs.


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Regenerating myelin for multiple sclerosis requires “contactin-1”

Contactin-1 is required for myelination of neurons damaged by multiple sclerosis  Read More...

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