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Like humans, some golden retrievers develop Duchenne muscular dystrophy (DMD), a hereditary muscle wasting condition that begins early in life. Using gene therapy, scientists were able to restore muscle function in dogs with the disease, according to a study published today (July 25) in Nature Communications.

"This case study introduces students to Duchenne muscular dystrophy (DMD) and its underlying genetics, cell biology, and some of the associated biochemical pathways.  DMD is an X-linked disorder characterized by progressive muscle weakness and wasting due to the absence of a protein called dystrophin, which in turn causes degeneration of skeletal and cardiac muscle. There is currently no established cure for this disease.  The case follows the progress of "Casey," an undergraduate student who has just declared her biology major and is interested in expanding her scientific understanding of the different fields of biology. The case is organized in three parts: genetics, cell biology, and biochemistry, each exploring DMD through its unique lens. Throughout the case, Casey is presented with multiple outlets of information, including class lectures, direct e-mail interaction with a professor, scientific journals and websites, from which she (and any student engaged with the case) gathers knowledge about DMD."

This directed case study was developed to introduce students to the CRISPR-Cas9 system for genome editing. CRISPR-Cas9 has made numerous headlines in both the scientific and popular press, and thus serves as an excellent model for learning current biotechnology and applying concepts from biology courses. After providing a general overview of CRISPR-Cas9, the majority of the case focuses on the clinical applications of the system as experienced by a carrier for the X-linked recessive allele underlying Duchenne Muscular Dystrophy. The case is structured so that students use a variety of popular and scientific sources (some of which may require a subscription to access-check with your institution), increasing in difficulty as they move through each part of the case. The goals are for students to learn the molecular mechanisms of CRISPR-Cas9, the benefits and limitations of the system, and the clinical applications of the technology. Open-ended questions are included to spark discussion of ethical considerations, societal impacts, and the overall implications of the technology.

The behavior of a person's genes doesn't just depend on the genes' DNA sequence - it's also affected by so-called epigenetic factors. Changes in these factors can play a critical role in disease.