Leber Congenital Amaurosis (LCA16) is a progressive vision loss disorder caused by point mutations in the KCNJ13 gene, which encodes an inward-rectifying potassium channel, Kir7.1.
Biomedical research has been revolutionized by the introduction of many CRISPR-Cas systems that induce programmable edits to nearly any gene in the human genome.
The lab of Kris Saha at the University of Wisconsin–Madison has developed an innovative combination of gene-editing tools and computational simulations that can be used to develop new strategies for editing genes associated with genetic disorders.
Waisman Center investigator David Gamm, MD, PhD, and Waisman affiliate Kris Saha, PhD, have published a proof-of-concept method to correct Best disease – an inherited form of macular degeneration that causes blindness, and that is …
Genome editors make targeted changes in the genome and hold great promise in both basic and translational research. Unfortunately, they often produce unwanted adverse effects, including genotoxicity, immune response, and reductions in cellular function.
Arezoo Movaghar earned her master’s degree in computer science and artificial intelligence. She built models based on the plentiful data found in medical records. So, when she came to UW–Madison as a PhD student and joined a research group, it surprised Movaghar to find out just how much data researchers in other fields collect.
Many scientists use animals to model human diseases. Mice can be obese or display symptoms of Parkinson’s disease. Rats get Alzheimer’s and diabetes. But animal models are seldom perfect, and so scientists are looking at a relatively new type of stem cell, called the induced pluripotent stem cell (iPS cell), that can be grown into specialized cells that become useful models for human disease.