gene therapy tagged posts

The ability to correct disease-causing genetic mistakes using genome editors holds great promise in medicine, but it is not without risk. When this type of “genetic surgery” is performed on DNA, for instance, there is always the danger of leaving permanent genetic scars that may even be heritable.

To alleviate this risk, researchers have experimented with gene editing processes on messenger RNA (mRNA), a central link between DNA and proteins that doesn’t carry the same risks because it doesn’t involve permanent changes to the DNA. But existing RNA editing tools have proven either too cumbersome to use or too toxic to human cells.

Yale researchers have developed a new—and safe—family of RNA-editing tools th...

Pain is meant to be a defense mechanism. It creates a strong sensation to get us to respond to a stimulus and prevent ourselves from further harm. But, sometimes injuries, nerve damage, or infections can cause long-lasting, severe bouts of pain that can make daily life unbearable.

What if there was a way to simply turn off pain receptors? UNC School of Medicine researchers Bryan L. Roth, MD, Ph.D., the Michael Hooker Distinguished Professor of Pharmacology, and Grégory Scherrer, PharmD, Ph.D., associate professor of cell biology and physiology and the UNC Neuroscience Center, have just proven that it is possible.

Using a tool designed by Roth in the early 2000s, the labs have created a new system that reduces acute and tissu...

A new gene therapy targeting the small brain region where dopamine neurons reside, the substantia nigra, substantially boosts the benefits of the drug levodopa in Parkinson’s. The therapy restored the ability of these neurons to convert levodopa to dopamine. Scientists also showed how damage to the powerplants inside dopamine-releasing neurons triggers Parkinson’s. The findings may help identify humans in the earliest stages of Parkinson’s disease, develop therapies to slow disease progression and treat late-stage disease.

In late-stage Parkinson’s disease, the drug levodopa becomes less effective in treating symptoms because of the inexorable loss of dopamine-releasing neurons...

The researchers used CRISPR and zinc fingers to repress NaV 1.7 and block pain signals in neurons in mice. [A.M. Moreno et al., Science Translational Medicine (2021)]

A gene therapy for chronic pain could offer a safer, non-addictive alternative to opioids. Researchers at the University of California San Diego developed the new therapy, which works by temporarily repressing a gene involved in sensing pain. It increased pain tolerance in mice, lowered their sensitivity to pain and provided months of pain relief without causing numbness.

The researchers report their findings in a paper published Mar. 10 in Science Translational Medicine.

The gene therapy could be used to treat a broad range of chronic pain conditions, from lower back pain to rare neuropathic pain disorders — co...