Gene therapy for a rare type of deafness is allowing deaf children and adults to hear for the first time, according to research results reported in April 2026.

Gene therapy is restoring hearing among kids with genetic deafness

Gene therapy for profound genetic deafness has been advancing in recent years, with multiple clinical trials enabling hearing at normal levels for the first time without hearing aids or cochlear implants. The therapies target hearing loss caused by mutations in the OTOF gene that codes for a protein called otoferlin. That protein normally plays a key role in relaying sound signals in synaptic transmission between the ear's inner hair cells and the auditory nerve.

Usher Syndrome, a rare genetic disorder, causes progressive hearing and vision loss, and people with the condition sometimes eventually become deaf and blind.

A planned clinical trial to treat a rare lysosomal storage disorder in utero has buoyed hopes of providing prenatal gene therapies, but it was still awaiting FDA approval to begin enrolling patients as of May 2026.

Scientists using gene therapy recently managed to regenerate retinal cells in the lab—a breakthrough for mammals that could potentially lead to future treatments for human retinal diseases.

Gene therapy, a hope of the Human Genome Project, has successfully treated a baby born with a rare urea cycle disorder that causes toxic ammonia level buildup.

In 2023, the FDA approved gene therapy for sickle cell disease, enabling one-time infusion bespoke treatments that use a patient's own stem cells to effectively cure a painful condition that disproportionately affects people of African American descent.

A single-gene mutation in the FOXC2 gene gave actress Elizabeth Taylor two rows of eyelashes.

More than 90% of American deaf children are born to hearing parents

The National Institute on Deafness and Other Communication Disorders have collated top statistics about hearing loss in the US including differences by age, racial groups, and other factors.

Gene therapy treats a disease by editing the disease's underlying DNA, replacing it with a functional variant.

How personalized gene-editing saved a baby's life

KJ Muldoon had a 50% chance of surviving a genetic mutation until doctors developed a one-of-a-kind gene-editing therapy. In an interview with one of the researchers behind the treatment, learn what it will take to make personalized genetic medicine possible for everyone.

Gene therapy can occur inside the body, but it can also be completed outside the body—with scientists treating cells in the lab, and then those corrected cells are transplanted back into the body.

In 2026, the FDA proposed a novel approach to expedite reviews of gene therapies for patients with rare genetic disorders such as cystic fibrosis, potentially bypassing the need for years-long clinical trials if approved.

Cochlear implants are raising questions about deaf identity and culture

The rise of cochlear implants, which are usually covered by insurance and can enable hearing for many patients, raises questions about what it means to be deaf, the effects on deaf individuals learning American Sign Language, and about deaf culture. Watch this video that discusses the controversies and spotlights a little girl who hears for the first time with her cochlear implant.

‘Hope for Healing’: The first-ever use of an in utero stem cell treatment

In 2023, researchers at UC Davis performed the first-ever fetal stem cell transplant, attempting to cure a case of spinal bifida prior to childbirth using stem cells derived from the placenta. The trial, and others including an effort to treat muscular dystrophy, highlight the potential power of stem cell therapy.

Big Ocean is breaking barriers as the world's first deaf K-pop boy band

The three-person boy band—whose members have different levels of hearing loss—use light systems, vibrations, and a mix of Korean and American Sign Languages, as well as International Sign (which isn't a whole language), to communicate and keep their dance routines in sync. In this interview, a band member with partial hearing describes their challenges.

The first neural devices were cochlear implants, designed to treat hearing loss

Neural interfaces connect with our nervous system to restore or enhance abilities, such as hearing or movement. These neuroprostheses have enabled the restoration of mobility, communication, and activities of daily living that might otherwise require a caregiver.

Eight British children have been born via mitochondrial replacement therapy

In July 2025, a decade after British lawmakers signed off on allowing three-parent IVF, scientists announced that eight healthy babies had already been born using this approach. The news was included in a pair of research articles published in a peer-reviewed journal that noted the oldest kids were already more than two years old, and that they were hitting developmental milestones as expected. (Some users may encounter a paywall.)

More than 5% of the world's population has disabling hearing loss

Disabling hearing loss is defined as hearing loss greater than 35 decibels in the better hearing ear. (For context, that means the sound of leaves rustling, at roughly 30 decibels, would be inaudible even to your better-hearing ear.) The World Health Organization's data suggests that more than 5% of the world's population has this level of loss requiring rehabilitation, which amounts to 430 million people—including 34 million children.

The first CRISPR-approved treatment was for sickle cell anemia in 2023

Victoria Gray was the first patient to receive a CRISPR-based therapy, which eliminated her symptoms and restored her health. The therapy's initial cost of $2.2M has kept it out of reach of many patients.

The first child born via mitochondrial replacement therapy was born in 2016

The healthy baby boy's mother carried genes for a rare mitochondrial disease called Leigh syndrome and after multiple miscarriages the family opted to try the controversial procedure. It was conducted by a US doctor in Mexico. The procedure is prohibited in the United States.

Carrier screening enables individuals without symptoms to see if they carry a gene for disease

Some conditions are autosomal recessive—meaning a person may carry a gene for that condition, but not have symptoms. Cystic fibrosis, sickle cell disease, and Tay-Sachs disease are examples of autosomal recessive diseases in which a person must inherit two copies of a nonfunctioning gene—one from each parent—to develop disease.

736,900 cochlear implants had been implanted by 2020 worldwide

In the United States, roughly 118,100 devices had been implanted in adults by that point and 65,000 in children. These estimated figures come from the US Food and Drug Administration, which first approved cochlear implants for adults experiencing hearing loss in the mid-1980s, and collected reports from cochlear implant manufacturers approved for the US market.

An ear doctor with hearing loss seeks to help those with hearing loss enjoy music

Alex Chern, an ear doctor who has hearing loss himself, dreams of restoring the ability to experience "all the richness and the beauty of the sound of music" for patients—not just functional hearing. Hearing aids amplify sound waves and aren't designed to work well with music that may have varied pitch and volume. Chern is studying how hearing loss and engaging with music may affect cognitive decline.

A first-of-its-kind clinical trial taps livers from gene-edited pigs to clean blood

The experimental dialysis-like treatment from United Therapeutics, eGenesis, and OrganOx, is designed to help people with liver failure. The therapy, which involves attaching the modified pig liver externally, filters a patient’s blood so their own livers can temporarily rest and perhaps heal.

See worldwide hearing loss by age group and country

This 2023 data from the Institute for Health Metrics and Evaluation shows the estimated number of people with complete hearing loss in different age groups per 100,000 people in the population. Individuals aged 70 or older make up the largest group with this type of hearing deficit.

The 'Armamentarium' is a set of new gene therapy tools—including viruses that could shuttle therapies into the body—designed to fight brain diseases.

Modern genetic testing stems from a 1950s discovery about the cause of Down syndrome

Scientists found that an additional copy of chromosome 21 causes the disease and developed an approach to stain chromosomes so they could be manually sorted and counted, which is called karyotyping. The breakthrough discovery by Marthe Gautier, a young French doctor and cytogenetics researcher, was appropriated by a male colleague, who listed himself as the lead author of the study reporting the finding.

The US FDA considered mitochondrial replacement therapy during 2014 hearings

An agency committee assessed if this approach could have undesirable ripple effects across multiple generations and whether a clinical trial involving this approach would be appropriate. As of 2026, the US has continued to prohibit this procedure though it is legal in other settings including the UK and Australia.

In a first, eye prosthesis has restored sight to macular generation patients

In a clinical trial, 27 of 32 participants with advanced macular degeneration regained enough vision to read books, food labels, and subway signs about a year after receiving a prosthetic retina device. This is the first device to restore functional vision to patients with incurable vision loss, though in this case they can only see in black and white, with no shades in between.

How the National Science Foundation helped cement the use of American Sign Language

Before the 1960s, deaf students were taught to read lips and pronounce words without signing, which was not considered a true language. After noticing that students nonetheless signed when communicating, American linguist William Stokoe conducted NSF-funded research that showed that signing met the definition of oral languages, thus establishing ASL.

Genetic engineering will change everything forever

Designer babies, the end of diseases, genetically modified humans that never age. Outrageous things that used to be science fiction are suddenly becoming reality. As scientists increasingly understand how specific genes cause disease, the possibility of engineering our genes to not only "fix" problems but enhance our natural ability grows.

Audism is institutionalized discrimination against deaf people

Promoting the idea of hearing-at-all-costs in medical and education settings has created division in the deaf community and influenced decisions about learning sign language.

OTC hearing aids are now available in the US and cheaper than prescription ones

The devices, available in recent years, are typically more affordable than prescription ones and are intended for people with mild to moderate hearing loss, yet surveys suggest people often don't feel comfortable choosing one on their own. The nonprofit Consumer Reports ranked available OTC options to help patients navigate the market.

Restoring vision in impaired patients via CRISPR

Since CRISPR was revealed as a gene editing tool in 2012, it has been on an accelerated track to real-world applications. Among other clinical demonstrations, doctors have now injected a CRISPR treatment directly into eye cells, helping vision-impaired patients see shapes and colors again.

Notch music therapy may filter out some tinnitus frequencies

Researchers in Germany have developed a smartphone app-based therapy for individuals with tinnitus associated with hearing loss. Some people with tinnitus can use this Notch app with their hearing aids to at least partially block sound frequencies matching their tinnitus pitch—turning down the volume of their tinnitus.

Tinnitus is a rare side effect of COVID-19

The infection can affect inner ear cells, and a review of the literature across multiple studies concluded that hearing loss occurred in 3.1% of patients with the virus and tinnitus in 4.5%.

A man with organ failure used AI to speed discovery of his miracle drug

David Fajgenbaum had a few months to live after a diagnosis with a rare form of Castleman's disease. By studying his blood, medical records, and broader literature, he was able to identify the cause of his condition and a treatment. He's been in total relapse for ten years.

Common genetic disorders

As research continues, it's clear that disease and genetics go hand in hand. Here's a selection of 50 National Human Genome Research Institute (NHGRI) articles providing basic information on genetic, orphan, and rare diseases caused by mutations in DNA sequences, whether inherited from parents or brought about by outside factors.