A new study presents an innovative treatment for deafness, based on the delivery of genetic material into the cells of the inner ear. The genetic material "replaces" the genetic defect and enables the cells to continue functioning normally.
The scientists were able to prevent the gradual deterioration of hearing in mice that had a genetic mutation for deafness. They maintain that this novel therapy could lead to a breakthrough in treating children born with various mutations that eventually cause deafness. The paper was published in EMBO Molecular Medicine.
Deafness is the most common sensory disability worldwide. According to the World Health Organization, there are about half a billion people with hearing loss around the world today, and this figure is expected to double in the coming decades. One in every 200 children is born with a hearing impairment, and one in every 1,000 is born deaf. In about half of these cases, deafness is caused by a genetic mutation. There are currently about 100 different genes associated with hereditary deafness.
"In this study we focused on genetic deafness caused by a mutation in the gene SYNE4 - a rare deafness discovered by our lab several years ago in two Israeli families, and since then identified in Turkey and the UK as well," the senior author reports. "Children inheriting the defective gene from both parents are born with normal hearing, but they gradually lose their hearing during childhood. The mutation causes mislocalization of cell nuclei in the hair cells inside the cochlea of the inner ear, which serve as soundwave receptors and are essential for hearing. This defect leads to the degeneration and eventual death of hair cells."
"We implemented an innovative gene therapy technology: we created a harmless synthetic virus and used it to deliver genetic material - a normal version of the gene that is defective in both the mouse model and the affected human families," says the lead author. "We injected the virus into the inner ear of the mice, so that it entered the hair cells and released its genetic payload. By so doing, we repaired the defect in the hair cells and enabled them to mature and function normally."
The treatment was administered soon after birth and the mice's hearing was then monitored using both physiological and behavioral tests. "The findings are most promising," says a collaborator on the study. "Treated mice developed normal hearing, with sensitivity almost identical to that of healthy mice who do not have the mutation."
The scientists are now developing similar therapies for other mutations that cause deafness.
https://aftau.org/news_item/scientists-at-tau-develop-new-gene-therapy-for-deafness/
https://www.embopress.org/doi/full/10.15252/emmm.202013259
http://sciencemission.com/site/index.php?page=news&type=view&id=publications%2Fneonatal-aav-gene&filter=22
A new gene therapy for deafness
- 1,430 views
- Added
Edited
Latest News
Mapping the molecular chang…
By newseditor
Posted 21 May
New mechanisms behind antib…
By newseditor
Posted 21 May
Blood-brain barrier from hu…
By newseditor
Posted 21 May
Genetic ancestry contribute…
By newseditor
Posted 21 May
Linked biological pathways…
By newseditor
Posted 21 May
Other Top Stories
Bacterial Effectors of Metformin Therapy
Read more
Why people gain weight as they get older
Read more
Increased interleukins alter gut microbiome to protect against type…
Read more
Microglial protein affects mitochondrial metabolism in high fat diet
Read more
Cognitive repair by diabetes drug only seen in females
Read more
Protocols
Efficient expansion and CRI…
By newseditor
Posted 21 May
Massively parallel in vivo…
By newseditor
Posted 20 May
Breast cancer-on-chip for p…
By newseditor
Posted 16 May
Methods for making and obse…
By newseditor
Posted 15 May
Mime-seq 2.0: a method to s…
By newseditor
Posted 13 May
Publications
Temporal dynamics of the mu…
By newseditor
Posted 21 May
Three concurrent mechanisms…
By newseditor
Posted 21 May
Parvalbumin interneuron cel…
By newseditor
Posted 21 May
Activation of GPR81 by lac…
By newseditor
Posted 21 May
Analysis of gene expression…
By newseditor
Posted 21 May
Presentations
Hydrogels in Drug Delivery
By newseditor
Posted 12 Apr
Lipids
By newseditor
Posted 31 Dec
Cell biology of carbohydrat…
By newseditor
Posted 29 Nov
RNA interference (RNAi)
By newseditor
Posted 23 Oct
RNA structure and functions
By newseditor
Posted 19 Oct
Posters
A chemical biology/modular…
By newseditor
Posted 22 Aug
Single-molecule covalent ma…
By newseditor
Posted 04 Jul
ASCO-2020-HEALTH SERVICES R…
By newseditor
Posted 23 Mar
ASCO-2020-HEAD AND NECK CANCER
By newseditor
Posted 23 Mar
ASCO-2020-GENITOURINARY CAN…
By newseditor
Posted 23 Mar