Advancing knowledge of human development, developmental disabilities, and neurodegenerative diseases.
The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) Consortium aims to accelerate the development of safer and more effective methods to edit the genomes of disease-relevant somatic cells in patients, even in tissues that are difficult to reach.
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.
Waisman Center investigator David Gamm, MD, PhD, and affiliate Krishanu Saha, PhD, are part of a team of scientists at the University of Wisconsin- Madison who were recently granted a major award from the National Institute of Health’s Somatic Cell Genome Editing Consortium (SCGE). This award will support research focused on developing quality control methods …
We present and characterize a robust method for rapid, scarless introduction or correction of disease-associated variants in hPSCs using CRISPR/Cas9. Utilizing non-integrated plasmid vectors that express a puromycin N-acetyl-transferase (PAC) gene, whose expression and translation is linked to that of Cas9, we transiently select for cells based on their early levels of Cas9 protein.
Waisman Biomanufacturing and Waisman affiliate investigators Randolph Ashton and Krishanu Saha will be part of a new engineering research center that will develop transformative tools and technologies for the consistent, scalable and low-cost production of high-quality living therapeutic cells. Such cells could be used in a broad range of life-saving medical therapies now emerging from …
Millions of people globally are at high risk for neurodegenerative disorders, infertility or having children with a disability as a result of the Fragile X (FX) premutation, a genetic abnormality in FMR1 that is underdiagnosed.
How much information can we extract from a five-minute recording of someone talking? Enough to tell whether that individual may be genetically predisposed to some health complications, according to researchers at the University of Wisconsin–Madison’s Waisman Center and Wisconsin Institute for Discovery. In a new study published this month in Scientific Reports, the researchers used …
Waisman Center researchers Anita Bhattacharyya and Xinyu Zhao are looking to make stem cells glow. That glow will tell them that they have successfully turned on a gene that is usually turned off in individuals with fragile X syndrome. Turning on this gene – called FMR1 – could be an important way to treat this …
