This activity builds on information presented in the short film Genes as Medicine. Students interpret actual pedigrees to determine the inheritance pattern of Leber congenital amaurosis (LCA), an inherited form of blindness.
Early last year, a little-known Chinese researcher turned up at an elite meeting in Berkeley, California, where scientists and ethicists were discussing a technology that had shaken the field to its core — an emerging tool for “editing” genes, the strings of DNA that form the blueprint of life
Scientists working on the frontiers of medicine fear the uproar over the reported births of gene-edited babies in China could jeopardize promising research into how to alter heredity to fend off a variety of disorders.
A Chinese researcher claims that he helped make the world’s first genetically edited babies — twin girls born this month whose DNA he said he altered with a powerful new tool capable of rewriting the very blueprint of life.
If true, it would be a profound leap of science and ethics.
Early, partial results from a historic gene editing study give encouraging signs that the treatment may be safe and having at least some of its hoped-for effect, but it’s too soon to know whether it ultimately will succeed.
U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly becoming an established form of medical care with no extraordinary risks.
Gene editing is getting fresh attention thanks to a successful lab experiment with human embryos. But for all the angst over possibly altering reproduction years from now, this technology already is used by scientists every day in fields ranging from agriculture to drug development.
This animation shows how mutations in an ion channel protein lead to the genetic disease cystic fibrosis. The animation also discusses how research on this protein has been used to develop treatments for the disease.