gene therapy tagged posts

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...

Scientists have developed a new gene-therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes.

Though the research was intended as a proof of concept, the experimental therapy slowed tumor growth and prolonged survival in mice with gliomas, which constitute about 80 percent of malignant brain tumors in humans.

The technique takes advantage of exosomes, fluid-filled sacs that cells release as a way to communicate with other cells.

While exosomes are gaining ground as biologically friendly carriers of therapeutic materials – because there are a lot of them and they don’t prompt an immune response – the trick with gene therapy is f...

Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe that the new technology can be likened to dramatically accelerating evolution from millions of years to weeks.

Several of the new revolutionary treatments that have been used clinically in recent years to treat complex diseases – such as spinal muscular atrophy and enzyme deficiency – are based on gene therapy.

With gene therapy, the genetic material is controlled or altered using biological drugs...