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CRISPR still has a long way to go before it can be used safely and effectively to repair-not just disrupt-genes in people. That is particularly true for most diseases, such as muscular dystrophy and cystic fibrosis, which require correcting genes in a living person because if the cells were first removed and repaired then put back, too few would survive. And the need to treat cells inside the body means gene editing faces many of the same delivery challenges as gene transfer-researchers must devise efficient ways to get a working CRISPR into specific tissues in a person, for example. CRISPR also poses its own safety risks. Most often mentioned is that the Cas9 enzyme that CRISPR uses to cleave DNA at a specific location could also make cuts where it's not intended to, potentially causing cancer.

Horn removal through gene editing is a win-win both for the animal and for the farmer, says Muir, who did not take part in this research. "These findings show that you can take highly desirable genes from animals and move them to other members of their species," he told Popular Science. "One could achieve the same results with natural breeding, but gene editing greatly speeds up the process, reducing the time it takes to accomplish the goal from centuries to years."

A new study shows that gene editing using CRISPR/Cas9 technology can work in rhesus monkey embryos. The results, published in the current issue of Human Molecular Genetics, open the door for pursuing gene editing in nonhuman primates as models for new therapies, including pharmacological, gene-, and stem cell-based therapies, says Keith Latham, animal science professor at Michigan State University and lead author of the study.

In our first animation of this series we learned how point mutations can edit genetic information. Here we see how duplication events can dramatically lengthen the genetic code of an individual. As point mutations add up in the duplicated region across generations, entirely new genes with new functions can evolve. In the video we see three examples of gene duplications resulting in new traits for the creatures who inherit them: the evolution of a venom gene in snakes, the evolution of leaf digestion genes in monkeys, and the evolution of burrowing legs in hunting dogs.

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.  

The war against malaria has a new ally: a controversial technology for spreading genes throughout a population of animals. Researchers report today that they have harnessed a so-called gene drive to efficiently endow mosquitoes with genes that should make them immune to the malaria parasite-and unable to spread it. On its own, gene drive won't get rid of malaria, but if successfully applied in the wild the method could help wipe out the disease, at least in some corners of the world. The approach "can bring us to zero [cases]," says Nora Besansky, a geneticist at the University of Notre Dame in South Bend, Indiana, who specializes in malaria-carrying mosquitoes. "The mosquitos do their own work [and] reach places we can't afford to go or get to."

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.

Chinese scientists say they've genetically modified human embryos for the very first time. The team attempted to modify the gene responsible for beta-thalassaemia, a potentially fatal blood disorder, using a gene-editing technique known as CRISPR/Cas9. Gene editing is a recently developed type of genetic engineering in which DNA is inserted, replaced, or removed. Here, experts weigh-in with ethical questions and considerations.

The uproar over the new ease and precision with which scientists can manipulate the DNA of living things has centered largely on the complicated prospect of editing human embryos. But with the federal government's approval last week of a fast-growing salmon as the first genetically altered animal Americans can eat, a menagerie of gene-edited animals is already being raised on farms and in laboratories around the world - some designed for food, some to fight disease, some, perhaps, as pets.

In a striking advance that helps open the door to organ transplants from animals, researchers have created gene-edited piglets cleansed of viruses that might cause disease in humans.

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.

Technical details aside, Crispr-Cas9 makes it easy, cheap, and fast to move genes around-any genes, in any living thing, from bacteria to people. "These are monumental moments in the history of biomedical research," Baltimore says. "They don't happen every day." Using the three-year-old technique, researchers have already reversed mutations that cause blindness, stopped cancer cells from multiplying, and made cells impervious to the virus that causes AIDS. Agronomists have rendered wheat invulnerable to killer fungi like powdery mildew, hinting at engineered staple crops that can feed a population of 9 billion on an ever-warmer planet. Bioengineers have used Crispr to alter the DNA of yeast so that it consumes plant matter and excretes ethanol, promising an end to reliance on petrochemicals. Startups devoted to Crispr have launched. International pharmaceutical and agricultural companies have spun up Crispr R&D. Two of the most powerful universities in the US are engaged in a vicious war over the basic patent.

Sharmila Nikapota, the mother of a child with a rare genetic disorder, has high hopes for gene editing. "For us this technology holds the unimaginable dream of a cure," she says. Her 13-year-old daughter Sohana has spent her entire life covered in painful blisters, the result of a condition called recessive dystrophic epidermolysis bullosa.

Geneticist Jennifer Doudna co-invented a groundbreaking new technology for editing genes, called CRISPR-Cas9. The tool allows scientists to make precise edits to DNA strands, which could lead to treatments for genetic diseases … but could also be used to create so-called "designer babies." Doudna reviews how CRISPR-Cas9 works - and asks the scientific community to pause and discuss the ethics of this new tool.

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.

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.

If the lab technique works in the field, it could offer a new way of stopping the biting insects from spreading malaria to humans, they say. The scientists put a new "resistance" gene into the mosquito's own DNA, using a gene editing method called Crispr. And when the GM mosquitoes mated - their offspring inherited the same resistance, PNAS journal reports. In theory, if these mosquitoes bite people, they should not be able to pass on the parasite that causes malaria.

Overview of genetic engineering and business assessment of scientific feasibility.  Good overview of past gene therapy attempts.

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.

We went to the U.S. Patent & Trademark Office to get the lowdown on the CRISPR gene-editing patent dispute. Read C&EN's coverage here: