(Press-News.org) By 2050, one in 10 individuals are expected to live with some form of hearing loss. Of the hundreds of millions of cases of hearing loss affecting individuals worldwide, genetic hearing loss is often the most difficult to treat. While hearing aids and cochlear implants offer limited relief, no available treatment can reverse or prevent this group of genetic conditions, prompting scientists to evaluate gene therapies for alternative solutions.
One of the most promising tools used in these therapies—adeno associated virus (AAV) vectors—has galvanized the hearing-loss community in recent years. Despite having already rescued hearing in neonatal animals with genetic defects, the vectors have yet to demonstrate this ability in fully mature or aged animal models. Since humans are born with fully developed ears, this proof-of-concept is necessary before testing the intervention in humans with genetic hearing loss.
A team of researchers from Mass Eye and Ear, a member of Mass General Brigham, recently became the first to successfully demonstrate AAV vector efficacy in aged animal models when they developed a mature mouse model with a mutation equivalent to a defective TMPRSS3 human gene, which typically results in progressive hearing loss. As reported in Molecular Therapy, researchers observed robust hearing rescue in the aged mice upon injecting the animals with an AAV carrying a healthy human TMPRSS3 gene.
“Our findings suggest that a virally mediated gene therapy, either by itself or in combination with a cochlear implant, could potentially treat genetic hearing loss,” said corresponding author Zheng Yi Chen, D.Phil., an investigator in the Eaton-Peabody Laboratories at Mass Eye and Ear. “This was also the first study that has rescued hearing in aging mice, which points to the feasibility of treating DFNB8 patients with DFNB8 even at an advanced age. The study also establishes the feasibility of other gene therapies in the aged population.”
in Molecular Therapy.
Gene therapy rescues hearing for the first time in aged mouse models
ELSE PRESS RELEASES FROM THIS DATE:
The mechanisms behind swallowing
Sensory cells in the vagus nerve can detect and locate food in the esophagus. Their signals help transport the food onward to the stomach. Signal failure leads to swallowing disorders, say a team led by Carmen Birchmeier at the Max Delbrück Center. They have published their findings in “Neuron.” Swallowing disorders can have many causes, and they occur more frequently in older people. But neurological diseases such as multiple sclerosis and Parkinson’s disease, and certain medications, can also prevent food moving normally from mouth to stomach. Possible consequences include malnutrition, weight loss, and dehydration. Now a team led ...
Life through rose-coloured glasses
Over thousands of years some animals have specialised to live in environments where the sun never shines: giant squid with eyes the size of volleyballs see even in the darkest depths while others, like cave-dwelling olms, have lost the functionality of their eyes completely. But for animals that do not live in these extremes, how do species manage a world that suddenly becomes dark? Lakes that become turbid from algal blooms, agricultural run-off, or other environmental pollutants represent common examples of environmental disturbances that can impact the visual scene that ...
Promising building blocks for photonic quantum simulators
Researchers at the Niels Bohr Institute have, collaborating with the University of Münster and Ruhr-Universität Bochum, developed new technology capable of processing the enormous amounts of information quantum systems generate. Deterministic single photon light sources, creating quantum bits at extreme rates and speed are now coupled to specially designed, integrated photonic circuits, capable of processing quantum information with adequate speed and quality without degrading the susceptible quantum states. This means that the first steps have been taken towards the development of photonic quantum devices that can, for example, ...
First measurements of hypernuclei flow at RHIC
UPTON, NY—Physicists studying particle collisions at the Relativistic Heavy Ion Collider (RHIC) have published the first observation of directed flow of hypernuclei. These short-lived, rare nuclei contain at least one “hyperon” in addition to ordinary protons and neutrons. Hyperons contain at least one “strange” quark in place of one of the up or down quarks that make up ordinary nucleons (the collective name for protons and neutrons). Such strange matter is thought to be abundant in the hearts of neutron stars, which are among the densest, most exotic objects in the universe. While blasting off to neutron stars to study ...
When the cell digests itself: How inherited neurodegenerative diseases develop
FRANKFURT. A tangle of pockets, tubes and sac-like membrane structures runs through the cells of humans, animals, plants and fungi: the endoplasmic reticulum, or ER for short. In the ER, proteins are manufactured, folded into their three-dimensional structure and modified, lipids and hormones are produced and calcium concentrations in the cell are controlled. In addition, the ER forms the basis for the cellular transport system, feeds misfolded proteins to intracellular disposal and renders toxins that have entered the cell harmless. In ...
Army funds two quantum-related projects at Pitt
The U.S. Army has awarded more than $5.7 million for two projects led by Michael Hatridge, associate professor of physics and astronomy in the Kenneth P. Dietrich School of Arts and Sciences. Both projects bring together a diverse group of researchers to overcome roadblocks in the field of quantum computing. A four-year, $2.67 million grant is aimed at the next generation of modular quantum computing systems. Hatridge and co-principal investigators Robert Schoelkopf of Yale University have each developed unique ...
Emergence of solvated dielectrons observed for the first time
Solvated dielectrons are the subject of many hypotheses among scientists, but have never been directly observed. They are described as a pair of electrons that is dissolved in liquids such as water or liquid ammonia. To make space for the electrons a cavity forms in the liquid, which the two electrons occupy. An international research team around Dr. Sebastian Hartweg, initially at Synchrotron SOLEIL (France), now at the Institute of Physics at the University of Freiburg and Prof. Dr. Ruth Signorell from ETH Zurich, including scientists ...
Networks in the dog brain
A study on canine brain networks reveals that during mammalian brain evolution, the role of the cingulate cortex, a bilateral structure located deep in the cerebral cortex, was partly taken over by the lateral frontal lobes, which control problem-solving, task-switching, and goal-directed behavior. The study relies on a new canine resting state fMRI brain atlas, which can aid in the analysis of diseases characterized by dysfunctional integration and communication among brain areas. Researchers interested in how dogs think can not only deduce it from their behavior, but they can also investigate their brain activity using fMRI (functional ...
Fractons as information storage: Not yet quite tangible, but close
Excitations in solids can also be represented mathematically as quasiparticles; for example, lattice vibrations that increase with temperature can be well described as phonons. Mathematically, also quasiparticles can be described that have never been observed in a material before. If such "theoretical" quasiparticles have interesting talents, then it is worth taking a closer look. Take fractons, for example. Perfect storage of information Fractons are fractions of spin excitations and are not allowed to possess kinetic energy. As a consequence, they are completely stationary and immobile. This makes fractons new candidates for perfectly secure information storage. Especially since ...
Defence lawyers face challenges accessing and reviewing digital evidence, study shows
Defence lawyers face numerous challenges accessing and reviewing evidence from phones and computers, a new study shows. Solicitors and barristers have reported their use of digital evidence can be restricted by limited or late access, large volumes of material, and tight turnaround times to secure legal aid funding and choose and instruct independent experts. The research calls for more clarity and transparency around the collection and analysis of digital evidence and the streamlining of the format and presentation of information. The current volume and diversity of digital evidence available escalates tensions, delays access to digital ...