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.

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.

Scientists may have discovered why gene-edited pig kidneys are rejected in humans

In November 2025, researchers studying how a brain-dead donor’s body responded to the transplantation of a genetically engineered pig kidney, published that they’ve identified immune system reactions that may explain why these pig organs get rejected. What’s more, they were able to reverse the rejections with an already available drug combination and the kidney functioned well. They also pinpointed a way to detect a potential rejection up to five days sooner.

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.

New eye implant clinical trial recruiting in 2026

University of Southern California researchers have launched a phase 2b clinical trial of a stem cell implant designed to restore vision in people with advanced dry macular degeneration. Patients will be randomized to receive the stem cell implant or a simulated procedure and placebo implant. This type of larger, second-stage clinical trial is designed to confirm efficacy and dosing and to look for side effects. It's one of the stages that precedes the US Food & Drug Administration approval of a device.

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 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.

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.

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.

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 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.

Using brain implants to restore vision

Several companies are attempting to create brain-computer interfaces that skip over retinas and optic nerves to allow visual input to go straight to the brain, potentially restoring some sight to the blind. Clinical trial findings suggest restored sight is sparse and manifests differently than a sighted person might expect, appearing as specks of "light" clusters called phosphenes to help differentiate one's surroundings.

Dozens of US research projects focus on the zebrafish's ability to grow new retina cells

The small, striped minnow can repair its optic nerve in just under a week. Scientists are interested in hacking its regenerative capacity to grow new retina cells to treat human eye disorders, including macular degeneration. In 2020, the US National Eye Institute had nearly 80 projects focused on zebrafish.

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

Targeting cholesterol metabolism may help address macular degeneration.

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.

US federal researchers are blocked from putting certain human cells in animals

In 2015, US policymakers put a halt to any government-funded work that would insert human stem cells into animals. The reasoning was that human cells might spread through the animal's body and perhaps to its brain, blurring the line between humans and nonhumans. The ban remains in place today even as gene-edited pig parts are being placed in humans.

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.

Chinese scientists transplanted a pig lung into a human being—but it had to be removed

In August 2025, Chinese scientists transplanted a pig lung into a brain-dead person, a common research step for this type of experimental work. Researchers used CRISPR technology to alter six proteins in the pig genes to make the organ seem less foreign to the human immune system. Within a day there were indications the organ wasn’t functioning well and that the person’s body was rejecting it. Scientists ended the experiment after nine days.

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

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

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.

Genetic engineering mice is one idea to reduce Lyme disease transmission

Wild white-footed mice are the main carriers of Lyme. A research team is hoping to genetically engineer mice, using CRISPR, to make the animals immune to Lyme. They're proposing a field trial where they would insert a gene with an antibody to Lyme infection to break the disease transmission cycle on a private island and then try it in Nantucket if that goes well. Simply killing the mice isn't an option because it would upend food webs. Unlike traditional vaccines, this "heritable immunization" would pass from one generation of mice to the next.

‘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.

In vitro gametogenesis: using induced stem cells to fertilize eggs

Using stem cells from skin and mouth cells could save hopeful parents time and money and spare egg donors from significant amounts of discomfort throughout the process. There are potential consequences, not just for infertility treatment, but for the ability to create customized cells of any type.

Synthetic biology can prevent extinction by creating create disease-resilient plants and animals

Through gene editing and the deployment of artificial cells, scientists can leverage learnings from the genetic sequencing of animals with known resistances to protect endangered species. Gene editing of certain species, such as mosquitoes, can also reduce their ability to spread diseases like malaria between humans.

Stem cell therapy has shown promise in treating spinal injuries in an early-stage clinical trial.

A clinical trial is underway in 2026 to help kidney transplant recipients avoid a lifetime of immunosuppressing drugs

This later-stage clinical trial, in Chicago and Taiwan, evaluates the efficacy and safety of a new cellular therapy that may be swapped in for immunosuppressing drugs among kidney transplant recipients. The study randomizes people to either receive standard immunosuppressants or this cellular therapy and fewer immunosuppressants. It only includes kidney transplant recipients receiving organs from live donors so transplant surgery timing won't hinge on the unpredictable schedule of performing surgery and follow-up care when a donor dies.

See different pig-to-human xenotransplantation possibilities

Xenotransplantation can include the insertion of any organ, tissue, cells or fluids from any nonhuman animal into a human. This US Food and Drug Administration graphic about pig-to-human applications shows different possibilities, including the exploration of using pig pancreatic cells for treating diabetes.

Indefinite cloning of some endangered species may not sustain their longevity because cloning success rates decrease as genetic mutations accumulate.

Early research suggests CAR-T therapy might treat scleroderma, reducing skin thickening

Clinical data from a couple patients in 2025 indicated that one experimental CAR-T therapy called rese-cel was able to genetically reprogram T cells to eliminate certain problematic immune B cells that were causing the condition. The therapy reduced skin thickening and allowed the patients to halt using immunosuppressive medications and steroids.

Researchers use the brain's ability to edit memories to explore memory deletion

Scientists have successfully implanted false memories in mice after studying how the editing process works in the brain. Beyond potentially deleting memories in those with PTSD, upcoming research may allow clinicians to forecast the onset of neurodegenerative diseases.

Limb regeneration research may lead to treatments for arthritis

For scientists pursuing the cure for arthritis, the holy grail of discoveries lies in how to regrow healthy cartilage inside human joints. While this feat has so far resisted our best scientific attempts, there are plenty of other human tissues—such as the liver and fingertips—that will successfully regenerate.

Photodynamic therapy is one current treatment for age-related macular degeneration

This laser-based treatment is used on advanced "wet" age-related macular degeneration patients, but it's now used less often than in the past because there are newer injection-based drugs to decrease abnormal blood vessel growth.

Move over gene editing, scientists can edit your epigenome

Instead of deleting genes, epigenetic editing modulates their activity. A new paper tests if it’s able to undo a genetic effect of early alcohol exposure.

Biotech is trying to boost drug production efficiency—using chicken eggs

A company called Neion Bio is genetically engineering birds in a bid to produce medical compounds, a herculean task that scientists have struggled with for decades. The US FDA has approved only one chicken-derived drug in the past—an expensive treatment for a rare liver disorder. (Some users may encounter a paywall.)

CRISPR is a gene-editing tool that lets scientists cut out and replace damaged DNA

One of the most significant advances in modern medical science, CRISPR is a molecular tool for making precise changes to a gene's DNA. The technology has already been approved for treating sickle-cell disease.

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.

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.