Victoria Gray, 34, of Forest, Miss., has volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the U.S. Meredith Rizzo/NPR hide caption.
But getting human cells to grow in another species is not easy. Nakauchi and colleagues announced at the 2018 American Association for the Advancement of Science meeting in Austin, Texas that they had put human iPS cells into sheep embryos that had been engineered not to produce a pancreas. But the hybrid embryos, grown for 28 days, contained very few human cells, and nothing resembling organs. This is probably because of the genetic distance between humans and sheep, says Nakauchi.
The research could eventually lead to new sources of organs for transplant, but ethical and technical hurdles need to be overcome.
Gene editing is advancing at a faster pace than most of us can keep up with. One significant recent announcement was gene editing tool CRISPR’s application to non-genetic diseases thanks to a new ability to edit single letters in RNA.
Even as CRISPR reaches milestones like this, scientists continue to find new uses for it to treat genetic conditions. The next one that will hit clinics is a CRISPR treatment for a form of blindness called Leber congenital amaurosis (LCA).
Having been approved by the FDA in December, the treatment will be the first of its kind to be trialed in the US.
In a milestone for forensic criminal investigators, a convicted killer received two life sentences on Wednesday for a 1987 double slaying after becoming the first person arrested through genetic genealogy to be found guilty at trial.
“The conviction and sentencing of William Earl Talbott II marks a new era for the use genetic genealogy for identifying violent criminals since it has now been tested and tried in a court of law,” geneology expert CeCe Moore told ABC News.
William Earl Talbott II was arrested in May 2018 and charged with aggravated murder for the Washington state cold case killings of 20-year-old Jay Cook and 18-year-old Tanya Van Cuylenborg, authorities said. A jury found Talbott guilty last month.
Patients are about to be enrolled in the first study to test a gene-editing technique known as CRISPR inside the body to try to cure an inherited form of blindness.
People with the disease have normal eyes but lack a gene that converts light into signals to the brain that enable sight.
The experimental treatment aims to supply kids and adults with a healthy version of the gene they lack, using a tool that cuts or “edits” DNA in a specific spot. It’s intended as a onetime treatment that permanently alters the person’s native DNA.
“I was wrong,” Church now admits.
A startup he cofounded, eGenesis, had made news for its ambitious plans to use CRISPR gene-editing technology to modify pigs so their organs could be safely transplanted into humans without being rejected. That could solve a critical shortage of human organs available for transplant.
But no human test has yet been carried out. Instead, the company is currently testing organs from its pigs in monkeys at Massachusetts General Hospital in Boston. The experiments are being led by the hospital’s chief of transplant surgery, James Markmann.
Arizona State University has been selected to participate in DARPA’s Epigenetic CHaracterization and Observation (ECHO) program. According to DARPA, the “ECHO program has two primary challenges: to identify and discriminate epigenetic signatures created by exposure to threat agents; and to create technology that performs highly specific forensic and diagnostic analyses to reveal the exact type and time of exposure.” (Epigenetic changes are chemical modifications that affect genes, altering their expression while leaving the genetic code intact. Epigenetic changes can occur as natural responses to the environment but can also signal exposure to toxic agents or disease pathogens.)
Epigenetics is coming into its own in the 21st century. DARPA describes the epigenome as “biology’s record keeper,” explaining that “though DNA does not change over a single lifetime, a person’s environment may leave marks on the DNA that modify how that individual’s genes are expressed. This is one way that people can adapt and survive in changing conditions, and the epigenome is the combination of all of these modifications. Though modifications can register within seconds to minutes, they imprint the epigenome for decades, leaving a time-stamped biography of an individual’s exposures that is difficult to deliberately alter.”
Sethuraman Panchanathan, ASU Knowledge Enterprise executive vice president and chief research and innovation officer, said the project fits with the university’s mission.
Recently, we had the opportunity to interview Professor George Church, a well-known geneticist and rejuvenation expert whom we have previously interviewed. Prof. Church’s company, Rejuvenate Bio, will be launching a clinical trial to test a rejuvenation therapy in dogs this fall.
In your recent paper on enabling large-scale genome editing, you talked about manipulating endogenous transposable elements with the help of dead Cas9 base editors. At Ending Age-Related Diseases, Andrei Gudkov spoke about the super mutagenic phenotype that arises from the expression of LINE1 reverse transcriptase. In this context, he mentioned the possibility of the retrobiome (as he referred to it) being the main driver of all types of cellular damage, which is consequently improperly addressed due to immunosenescence. Do you share his views on the contribution of LINEs and SINEs in aging? If not, why?
Yes. That is one of the reasons why we explored the tech for editing of repeats. We are now extending this to the germline engineering of repeats.
HELLO! https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6025786/
With approximately 40 million adults across the United States experiencing anxiety each year, scientists and researchers have dedicated their careers to trying to better understand this condition. Despite this work, we are still somewhat unclear on what actually causes this condition to occur.
