USC Institute for Genetic Medicine The USC Institute for Genetic Medicine (IGM) is a place where researchers work closely together in a shared space to approach problems of the highest impact to human health. The IGM houses the offices and laboratories of a group of leading faculty members from the University of Southern California (USC). IGM scientists, engineers and clinicians take a multidisciplinary approach using molecular, genetic, omics, and computational technologies in human and genetic models. The IGM is administered as a department reporting to the Dean of the Keck School of Medicine of USC. It is part of the USC Health Sciences Campus, located on the east side of Los Angeles. The IGM comprises 21 faculty members. The department is an important research training ground for USC, and typically supports about 50 trainees at any one time. Including scientific and administrative staff, the total number of people working within the IGM hovers around 110 individuals. IGM research is funded by federal, state, and industry-sponsored grants, as well as gifts from donors and benefactors. The major sponsor is the National Institutes of Health (NIH). The Center for Applied Molecular Medicine (CAMM) within the IGM holds a Physical Sciences in Oncology … Continue reading →

Researchers at UT Southwestern Medical Center have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich's ataxia, a neurological disease for which there is currently no cure. Friedreich's ataxia results from modifications to DNA sequences that prevent cells from producing enough of a needed protein called frataxin. The lack of frataxin can result in a variety of problems that include loss of muscle control, fatigue, vision or hearing impairment, slurred speech, and serious heart conditions. Using synthetic RNA or DNA, researchers have identified a way to allow normal frataxin production to resume. "The synthetic DNA or RNA prevents the mutant sequence from bending back and blocking the frataxin gene. This action activates the frataxin gene, which then makes frataxin RNA and protein at normal levels," said Dr. David Corey, Professor of Pharmacology and Biochemistry. "In addition, our approach is selective for targeting the frataxin gene FXN and does not affect other genes." In contrast to the CRISPR genomic editing technique, which requires modifications to genes, the molecules in this study are synthetic. The DNA and RNA belong to classes of molecules that already are being used clinically, making development of a new therapy more straightforward, said Dr. … Continue reading →

Practice Info The Department of Genetic Medicine at Weill Cornell Medicine is a highly specialized form of personalized medicine that involves the introduction of genetic material into a patients cells to fight or prevent disease. This experimental approach requires the use of information and data from an individual's genotype or specific DNA signature, to challenge a disease, select a medication or its dosage, provide a specific therapy, or initiate preventative measures specifically suited to the patient. While this technology is still in its infancy, gene therapy has been used with some success and offers the promise of regenerative cures. As none of New York's premier healthcare networks, Weill Cornell Medicine's genetic research program includes close collaborations with fellow laboratories such as Memorial Sloan Kettering Cancer Center for stem cell projects, Weill Cornell Medical College in Qatar and Hamad Medical Corporation in Doha, Qatar and Bioinformatics and Biostatistical Genetics at Cornell-Ithaca. Department of Genetic Medicine Services Our translational research program includes many projects in the fields of genetic therapies and personalized medicine, and we arestudying gene therapy for a number of diseases, such as combined immuno-deficiencies, hemophilia, Parkinson's, cancer and even HIV using a number of different approaches. Patients interested in … Continue reading →