Many basic and clinical researchers are testing the potential of a simple and efficient gene editing approach to study and correct disease-causing mutations for conditions ranging from blindness to cancer, but the technology is constrained by a requirement that a certain short DNA sequence be present at the gene editing site.

The gene-editing tool CRISPR has been used for the first time inside the body of an adult patient, in an attempt to cure a form of blindness. The treatment: According to the Associated Press, doctors dripped just a few drops of a gene-editing mixture beneath the retina of a patient in Oregon who suffers from Leber congenital amaurosis, a rare inherited disease that leads to progressive vision loss.

The tool was used in an attempt to treat a patient's blindness. It may take up to a month to see if it worked.

What would the implications be if decoding your genes cost less than a pair of designer jeans? We might soon find out after a Chinese company claimed it can sequence the human genome for $100. The speed at which the price of genetic sequencing has fallen has been astonishing, from $50,000 a decade ago to roughly $600 today. For a long time, the industry saw the $1,000 genome as the inflection point at which we would enter the genomic age—where getting a read out of your DNA would be within reach for huge swathes of the population.

Duchenne muscular dystrophy (DMD) is one of the most common and most devastating muscular diseases, greatly reducing patients’ quality of life and life expectancy. Now, researchers in Germany have managed to use the CRISPR gene-editing tool to correct the condition in pigs, bringing the treatment ever closer to human trials.

Scientists from the University of Pennsylvania just published the results of the first U.S. trial of CRISPR-edited cells in cancer patients — and they’re very encouraging. In April 2019, UPenn confirmed that a team of its researchers had officially begun testing CRISPR-edited cells in humans.

Scientists report positive safety and durability results from the first U.S. test of CRISPR gene editing in human patients with advanced cancer. Technology Networks spoke to the researchers to find out more.

Microsoft co-founder Bill Gates has been working to improve the state of global health through his nonprofit foundation for 20 years, and today he told the nation’s premier scientific gathering that advances in artificial intelligence and gene editing could accelerate those improvements exponentially in the years ahead.

HIV has no cure. It’s not quite the implacable scourge it was throughout the 1980s and 1990s, thanks to education, prophylactics, and drugs like PrEP. But still, no cure.The same ability, though, could be HIV’s undoing. All because of CRISPR. You know, CRIPSR: the gene-editing technique that got everyone really excited, then really sceptical, and now cautiously optimistic about curing a bunch of intractable diseases.

What now? Researchers in Portland, Oregon have used a powerful gene editing tool called CRISPR to create genetically modified human embryos, MIT Technology Review reported on Wednesday in an exclusive. This is the first confirmed case of the gene editing of human embryos in the US, and gets scientists closer to the ability to create genetically engineered humans.

Gene-edited human embryos have offered a glimpse into the earliest stages of development, while hinting at the role of a pivotal protein that guides embryo growth. The first-of-its-kind study stands in contrast to previous research that attempted to fix disease-causing mutations in human embryos, in the hope of eventually preventing genetic disorders. Whereas those studies raised concerns over potential ‘designer babies’, the latest paper describes basic research that aims to understand human embryo development and causes of miscarriage.

In September, the U.S. Department of Agriculture gave the green light to a version of the plant Camelina sativa, an important oilseed crop that had been genetically engineered using CRISPR to produce enhanced omega-3 oil. What was interesting about this approval was that the USDA did not ask that the inventors of the plant endure the usual regulatory hoops required to sell biotech crops. The next month, a drought-tolerant soybean variety developed with CRISPR also got a quick pass from the USDA.

Scientists have successfully tweaked the DNA in human heart cells to correct mutations that cause a deadly disease. If the gene-editing technique is proven safe, it could permanently cure children with a genetic disorder that leaves them wheelchair-bound by their early teens. The genetic disease targeted in this study is called Duchenne muscular dystrophy (DMD), a disorder that affects about one in 5,000 males and causes the progressive wasting away of skeletal and heart muscles.

Author summary The causative agent of malaria, Plasmodium, has to complete a complex infection cycle in the Anopheles gambiae mosquito vector in order to reach the salivary gland from where it can be transmitted to a human host. The parasite’s development in the mosquito relies on numerous host factors (agonists), and their inhibition or inactivation can thereby result in suppression of infection and consequently malaria transmission.

Right before I turned five, I went to the eye doctor for the first time. I even wrote about it in my kindergarten journal: “I am going to get galassis! I am so aixidad!” (I was a complicated speller.) At that appointment, I was diagnosed with vitelliform macular dystrophy, a degenerative eye disease also known as “Best disease.” Which doesn’t make a ton of sense because Best disease causes my eyesight to get worse and worse over time. It’s a lot like age-related macular degeneration, but it’s genetic, and my vision could deteriorate at any time, not just when I’m 80.

A scientific journal has retracted a controversial paper, published last year, that suggested the gene-editing tool CRISPR was a genome wrecking ball. In the retracted study, researchers sought to use CRISPR in mice to correct a mutation that causes blindness. They successfully fixed the genetic error but reported that CRISPR inadvertently made more than a thousand other changes—potentially harmful ones—in the animals’ DNA.

Biomedical engineers have used a CRISPR/Cas9 genetic engineering technique to turn off a gene that regulates cholesterol levels in adult mice, leading to reduced blood cholesterol levels and gene repression lasting for six months after a single treatment. This marks the first time researchers have delivered CRISPR/Cas9 repressors for targeted therapeutic gene silencing in adult animal models. The study appears in Nature Communications.

A Salk Institute team transplanted liver cells into mouse models of hemophilia B, finding that the treatment restored their ability to form blood clots for a year. The hope is that this one-and-done treatment could replace the frequent injections of clotting factors that are currently used to treat the inherited blood disorder.

It's so small it can’t be seen with the naked eye, but research is showing that CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is bringing sight to the blind.

Scientists from China and the USA report the use of CRISPR/Cas9 to develop a variety of rice producing 25-31% more grain than traditional breeding methods.