Researchers have found differences in how Alzheimers disease originates and develops in mice and humans. They determined which gene causes Alzheimers in humans and have developed a fix for it. Dementia and Alzheimer’s disease aren’t the same. Dementia is an overall term used to describe symptoms that impact memory, performance of daily activities, and communication …
In a landmark study that could lead to new therapies for sickle cell anaemia and other blood disorders, UNSW Sydney-led researchers have used CRISPR gene editing to introduce beneficial natural mutations into blood cells to boost their production of foetal haemoglobin. The research solves a 50-year-old mystery about how these mutations – which are naturally …
China has at least eight CRISPR gene therapy clinical trials to treat various forms of cancer. CRISPR is being tested as a treatment for cancers of the lung, bladder, cervix and prostate. Clinicaltrials.gov lists all of the clinical trials in the world. Only one CRISPR cancer study has been approved in the United States, and …
Researchers review the pioneering work that led the gene therapy field to its current state, describe gene-editing technologies that are expected to play a major role in the field’s future, and discuss practical challenges in getting these therapies to patients who need them. Above- Three essential tools for human gene therapy. AAV and lentiviral vectors …
Crispr/Cas9 is a riboprotein complex composed of a short strand of RNA and an efficient DNA-cutting enzyme. Crispr/Cas9 sometimes cuts in the wrong places. And it has no off-switch. Epigenetic Editing Other proteins—like ones that activate gene expression—can be combined with a crippled Cas9, letting them toggle genes on and off (sometimes with light or …
Researchers deliver CRISPR-Cas9 directly into the inner ear hair cells of mice, preventing hearing loss in animal model of genetic progressive deafness. Above – Credit : Gao et al./Nature 2017. Confocal microscopy images of mouse cochlea; hair cells in green. (Left) An untreated cochlea from a mouse with the Tmc1 mutation, displaying hair cell loss. …
University of California, Berkeley scientists have for the first time used CRISPR-Cas9 gene editing to disable a defective gene that causes amyotrophic lateral sclerosis, or Lou Gehrig’s disease, in mice, extending their lifespan by 25 percent. The therapy delayed the onset of the muscle wasting that characterizes the disease, which results in progressive weakness and …
Researchers have identified and characterized a new type of RNA protein called THOR that plays a role in cancer development — and could serve as a target for drug development. Researchers at the University of Michigan Comprehensive Cancer Center uncovered a novel gene they named THOR while investigating previously unexplored regions of the human genome …
Scientists have been able to reverse heart failure in pigs by delivering a new gene to the heart that makes it better at pumping blood and supplying the body with oxygen. The goal is to reverse heart failure with a single injection. This time around, Dr. Hajjar said, he and his team have reengineered the …
For the first time, researchers have infused a person’s blood with gene editing tools, aiming to treat his severe inherited disease (
James Clement has collected blood, skin or saliva from supercentenarians in 14 states and seven countries over a six-year period. Lifestyle and luck, it seems, still factor heavily into why people live into their 90s and 100s. However, supercentenarians are more uniformly healthy than centenarians in their final months and years. Rather than having won …