GENE THERAPY breakthrough. Start of a new era for gene therapies as 18mt old UK toddler regains hearing
A rare genetic condition, auditory neuropathy, causing the disruption of nerve impulses travelling from the inner ear to the brain, due to a variation in a single gene, The OTOF gene. The gene produces a protein called otoferlin, needed to allow the inner hair cells in the ear to communicate with the hearing nerve. “Mind-blowing” results, within four weeks of having the gene therapy infusion to her right ear, child responded to sound. The first patient treated in a global gene therapy trial, youngest child to receive this type of treatment in the world . App. 20,000 people across the UK, Germany, France, Spain, Italy and UK and are deaf due to a mutation in the OTOF gene.
GENE THERAPY breakthrough. An electrifying discovery may help doctors deliver more effective gene therapies. Short electric pulses caused the liver cells to take in more than 40 times the amount of gene therapy material
In an effort to improve delivery of costly medical treatments, a team of researchers in electrical engineering has developed a stimulating method that could make the human body more receptive to certain gene therapies.It improve efficiency of medical treatments, electrical engineering researchers at the University of Wisconsin-Madison have exposed liver cells to short electric pulses — the liver cells toke in more than 40 times the amount of gene therapy material compared to cells that were not exposed to pulsed electric fields. The method could help reduce the dosage needed for these treatments, making them much safer and more affordable. One of the bottlenecks in gene therapy, however, is getting the right dose of genetic material into the target cells. The UW-Madison research suggests that applying a moderate electric field, which left no lasting damage to the cells that received it, could help in creating more effective therapies.
Date:April 30, 2024
Source:University of Wisconsin-MadisonSummary:
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