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1105 results for "otof gene"
A seven-year-old girl born deaf had conversations with her mother just four months after one injection into her inner ear. All 10 trial participants showed measurable hearing improvement without implants or devices.
Melbourne researchers developed an AI tool that creates molecular "off switches" for CRISPR gene editing in just 8 weeks. The breakthrough could help make gene therapy treatments safer and more accessible for patients with genetic diseases.
Australian researchers developed an AI system that makes gene editing safer by protecting healthy genes during treatment. The breakthrough could transform how doctors treat genetic diseases like cystic fibrosis.
Children born completely deaf are hearing whispers for the first time thanks to an experimental gene therapy that has shown remarkable success in the largest study of its kind. The treatment could become the first FDA-approved gene therapy for deafness within months.
After decades of skepticism, researchers have perfected a gentler way to control genes using epigenetic editing. This breakthrough lets them dial gene expression up or down without changing DNA itself, opening doors to safer treatments and smarter crops.
Scientists used AlphaFold AI to redesign gene-editing proteins, making them dramatically safer by nearly eliminating unwanted DNA changes. This breakthrough could help life-saving gene therapies reach more patients with fewer risks.
Scientists built an AI that designs synthetic DNA switches capable of turning genes on and off in specific cells, potentially transforming how we treat diseases like leukemia. In lab tests, these AI-generated sequences actually outperformed natural DNA at reactivating a protective gene in leukemia cells.
A groundbreaking gene therapy trial has achieved what doctors call a "functional cure" for sickle cell disease, eliminating painful crises in 27 out of 28 patients. The one-time treatment uses gene editing to fix the blood disorder that has killed patients decades too soon.
Children born with a specific type of genetic hearing loss can now hear their parents' voices and dance to music, thanks to the FDA's approval of the first-ever gene therapy for inherited deafness. The one-time treatment helps patients avoid cochlear implants and restores natural hearing.
A seven-year-old girl born deaf had everyday conversations with her mother just four months after a single injection delivered a missing gene to her inner ear. Ten patients treated in a groundbreaking trial can now hear sounds they never could before.
A groundbreaking gene therapy trial has restored hearing in 90% of people born profoundly deaf, with improvements lasting over two years. The treatment worked across ages from infants to adults, offering new hope beyond cochlear implants.
Children born deaf from a genetic mutation can now hear naturally thanks to a groundbreaking gene therapy just approved by the FDA. The one-time treatment restores a missing protein in the inner ear, allowing sound signals to reach the brain.
The FDA just approved the first gene therapy to treat genetic hearing loss, giving hope to dozens of families each year whose babies are born deaf. Even better? The company is making it free for all U.S. patients.
Children born deaf are now hearing and speaking for the first time, thanks to a breakthrough gene therapy that reversed a rare inherited form of deafness. The treatment's effects have lasted up to 2.5 years, the longest follow-up yet reported.
Children born deaf are now hearing their mother's voice for the first time, thanks to a groundbreaking gene therapy that restores natural hearing. The FDA just approved the treatment, and the company is offering it completely free across the United States.
Chinese scientists successfully treated children born deaf by delivering a missing gene directly into their inner ear cells. All patients under age 3 can now hear everyday conversations after a single treatment.
Scientists have developed a new gene editing approach that spreads healing changes from cell to cell, dramatically increasing its reach in the body. This breakthrough could transform treatment for countless genetic diseases that were previously too difficult to address.
Serenity Cole, 18, spent years in constant pain from sickle cell disease. Now she's pain-free thanks to a revolutionary payment deal where drug companies only get paid if their $2-3 million gene therapies actually work.
Scientists tested a one-time gene therapy that lowered cholesterol up to 62% in patients, potentially replacing daily pills and injections with a single treatment. The breakthrough could transform how millions manage heart disease risk.
University of Virginia researchers used gene editing to fix the DNA mutation causing a deadly form of epilepsy, potentially opening the door to cures for inherited brain disorders. The technique eliminated seizures in lab mice and improved their movement and cognitive function.
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