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More than 400 scientists, bioethicists, and historians from 20 countries on 6 continents have gathered this week in Washington, DC, for the Human Gene Editing Summit. The attendees are a veritable who's who of genome editing: Jennifer Doudna of the University of California, Berkeley, Emmanuelle Charpentier of Max Planck Institute for Infection Biology, and Feng Zhang of the Broad Institute of MIT and Harvard-the three discoverers of the CRISPR-Cas9 system's utility in gene editing-plus dozens of other big names in genome science. Cal Tech's David Baltimore along with the heads of the four national societies hosting the meeting (US National Academy of Sciences, US National Academy of Medicine, Chinese Academy of Sciences, and the U.K.'s Royal Society) provided opening remarks on Tuesday (December 1). And as I sat stage right in the NAS auditorium, I noticed the unmistakable rear profile of Harvard Medical School's George Church three rows in front of me. Church was scheduled to speak at a session later that afternoon about the application of CRISPR and other new precision gene editing techniques to the human germline-a hot-button topic since April, when a Chinese group published it had successfully modified the genomes of human embryos, and the National Institutes of Health (NIH) said it would not fund such research. Then in September, the U.S./U.K.-based Hinxton Group, an international consortium of scientists, policy experts, and bioethicists, said it supported the use of genetic editing in human embryos for limited applications in research and medicine.  

An international group of scientists meeting in Washington called on Thursday for what would, in effect, be a moratorium on making inheritable changes to the human genome. The group said it would be "irresponsible to proceed" until the risks could be better assessed and until there was "broad societal consensus about the appropriateness" of any proposed change. The group also held open the possibility for such work to proceed in the future by saying that as knowledge advances, the issue of making permanent changes to the human genome "should be revisited on a regular basis."

This animation depicts the CRISPR-Cas9 method for genome editing - a powerful new technology with many applications in biomedical research, including the potential to treat human genetic disease. Feng Zhang, a leader in the development of this technology, is a faculty member at MIT, an investigator at the McGovern Institute for Brain Research, and a core member of the Broad Institute. Further information can be found on Prof. Zhang's website at http://zlab.mit.edu .

An environmentally dependent method to excise particular genes and eliminate genetically modified organisms (GMOs) if they leave the lab, published this week (May 19) in Nature Communications, uses an inducible CRISPR/Cas9 genome-editing system to snip out vital pieces of the E. coli genome.

Over the last decade, as DNA-sequencing technology has grown ever faster and cheaper, our understanding of the human genome has increased accordingly. Yet scientists have until recently remained largely ham-fisted when they've tried to directly modify genes in a living cell. Take sickle-cell anemia, for example. A debilitating and often deadly disease, it is caused by a mutation in just one of a patient's three billion DNA base pairs. Even though this genetic error is simple and well studied, researchers are helpless to correct it and halt its devastating effects.

Tweaking the human genome with current and future gene-editing tools could lead to sophisticated treatments and prevention strategies for disease. The promise of those applications is reason enough to move forward with such work in the lab and clinic, albeit cautiously, the dozen scientists and bioethicists who organized the International Summit on Human Gene Editing said today after three days of deliberation and presentations in Washington, D.C.

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.

A British researcher has received permission to use a powerful new genome editing technique on human embryos, even though researchers throughout the world are observing a voluntary moratorium on making changes to DNA that could be passed down to subsequent generations.

Three years ago, Dr. Doudna, a biochemist at the University of California, Berkeley, helped make one of the most monumental discoveries in biology: a relatively easy way to alter any organism's DNA, just as a computer user can edit a word in a document.

In this video Paul Andersen explains how the CRISPR/Cas immune system was identified in bacteria and how the CRISPR/Cas9 system was developed to edit genomes.

Following on the achievements of Chinese researchers, scientists in the United States have used CRISPR to manipulate the genomes of viable human embryos, MIT Technology Review reported yesterday (July 26). The work, not yet published, reportedly corrected defective genes from sperm donors in dozens of embryos, which were allowed to grow for several days.

In a first, scientists led by Kamel Khalili, director of the Comprehensive NeuroAIDS Center at Temple University, report in the journal Gene Therapy that they have for the first time successfully eliminated HIV genes from the genomes of mice and rats infected with the virus.

Current CRISPR-based screens often mutate the beginning of a gene, which sometimes results in the expression of a functional protein variant. To circumvent this problem, researchers at Cold Spring Harbor Laboratory (CSHL) designed CRISPR guide RNAs that would mutate the portion of a gene encoding a domain on the surface of the protein where a small molecule could bind to alter the protein's function. The team had previously identified such a binding pocket on the protein BRD4, and a small molecule inhibitor that binds in the pocket is an effective leukemia treatment.