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.

See how a novel gene editing technique called base editing can create changes in a single DNA base, using a more precise targeting approach than CRISPR-Cas9.

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

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.

Epigenetic editing—altering gene expression by removing or adding chemical groups on DNA—is making significant strides in the lab, even lowering cholesterol in monkey experiments by about 70%. (Free w/email.)

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.

CRISPR allows scientists to cut out and replace faulty or unwanted DNA, which can help treat genetic disorders, such as sickle cell disease, and create genetically modified organisms, such as allergen-free foods.

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.

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 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.

See this list of FDA-approved gene therapies, including offerings for inherited conditions as well as some forms of cancer.

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

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.

While humans have been genetically modifying plants for millennia by selectively breeding organisms with certain traits, gene-editing tools like CRISPR have further enhanced plants, conferring resistance to drought, pests, and diseases.

CRISPR-Cas9 enables precise genome editing using a simple RNA-guided system

Jennifer Doudna's research uncovered how bacteria use the tool as an immune defense by cutting viral DNA. She and Emmanuelle Charpentier received the 2020 Nobel Prize in chemistry for showing how this mechanism could be engineered to target and edit genes in living cells.

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.

CRISPR can't fix faulty mitochondria

Gene editing via CRISPR could theoretically fix many issues in biology, but editing the mitochondrial genome has remained challenging. In recent years, there has been some progress reaching these cellular powerhouses. (Some users may experience a paywall.)

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.

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.

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.

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.

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

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.

Astronauts have repaired genetic damage in space using CRISPR—a molecular tool that can make precise changes to DNA and is used in single-celled organisms to attack invasive viruses.

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

Visualize how CRISPR-Cas9—a type of immune system used by single-celled organisms to attack invasive viruses—targets and makes precise changes to a gene's DNA.

Aurea is the first US baby born following polygenic analysis

The child, born in May 2020, was selected by her parents to optimize for certain health qualities based on an analysis of her multifactorial genes by a New Jersey-based company called Genomic Prediction. Her embryo was selected for implantation based on polygenic risk scores for heart disease, diabetes, and cancer.

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.)

Explaining CRISPR at five levels of technical detail

CRISPR is a new area of biomedical science that enables gene editing and could be the key to eventually curing diseases like autism or cancer. WIRED challenges biologist Neville Sanjana to explain this concept to 5 different people; a 7-year-old, a 14-year-old, a college student, a grad student and a CRISPR expert.

A deeper (nontechnical) dive into how CRISPR works

Extra crispy? No, it's CRISPR, but this gene-editing technology is "extra" in the sense of being momentous and destined to change everything. Through CRISPR, scientist exploit a quirk in bacteria immune systems, allowing them to potentially, and quickly, control genes, remove undesirable traits, and add preferred ones. The possibilities and applications are endless.

CRISPR turns 10 years old

When Dr. Jennifer Doudna and colleagues released a paper in 2012 demonstrating the ability to harness the cell's natural immunity machinery to edit DNA, it received little fanfare. A decade later, the gene editing technology has led to innovations in medicine, evolution, and agriculture—and raised profound ethical questions about altering human DNA.

Genetic engineering has accelerated millennia of altering various crops' genes

Although farmers have long cross-bred crops to obtain plants with specific colors, sizes, and other characteristics, these processes took significant time. Today, genetic modification in agriculture involves scientists altering an organism's DNA for various purposes, including enhancing pathogen resistance, removing allergens, and delaying spoilage.

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.

‘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 2025, researchers in the UK began working on developing artificial DNA

The Synthetic Human Genome Project aims to one day construct human DNA, molecule by molecule, in the hopes of creating disease-resistant cells and synthetic chromosomes for study. However, critics warn it may be misused to create enhanced or modified humans.

Advances in agriculture are being powered by CRISPR to boost carbon capture

Researchers are using CRISPR to test thousands of plant gene edits, aiming to speed up breeding and maximize carbon retention in soil. By engineering drought tolerance, they also aim to increase crop yields and expand the environments where crops can grow.

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.

The first newborn screen for diseases was developed in 1961.

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.

Gut bacteria are evolving and can acquire as many as 100 new genes per year, on average.

A physician with a rare and deadly disease saved his own life by identifying an existing medication that he repurposed for his condition, and now he's using AI to try to do the same for other patients.

CRISPR technology is removing allergens from common foods

Proteins responsible for allergies to eggs, milk, wheat, peanuts, and other foods can be genetically edited out of foods to reduce the risk of life-threatening reactions. The technique has also been used to produce caffeine-free coffee beans, spicier tomatoes, allergen-free pets, and more nutritious fish.

Genetic drug matching, called pharmacogenomics, can help avoid dangerous drug reactions

Certain gene variants can affect how quickly or slowly medications are broken down in the body, affecting drug effectiveness and the risk of drugs failing. Such testing is often not covered by insurance companies though research indicates if these tests were performed for more than six drugs across the US population, avoided ailments would likely climb into the hundreds of thousands. (Some users may encounter a paywall.)

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.

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.)

Drs. Jennifer Doudna and Emmanuelle Charpentier discuss CRISPR

Jennifer Doudna and Emmanuelle Charpentier's were awarded the 2020 Nobel Prize for research focused on understanding and adapting the CRISPR-Cas9 system to precisely edit DNA sequences in other organisms. Doudna has indicated that the system can potentially treat genetic forms of blindness and high cholesterol.

3-parent baby technique fuels worries of future 'designer babies'

Some critics of this approach worry that any genetic manipulation will both culturally and technologically normalize the idea of "designer babies"—embryos that are manipulated or selected based on physiological characteristics including height, eye color, athletic ability, and intelligence.

Many of the new prenatal genetic tests for rare genetic conditions are inaccurate

Established screening tests for Down syndrome in utero are reliable and accurate. But a New York Times investigation into expanded prenatal genetic tests for very rare diseases—much rarer than Down syndrome—found that more than 80 percent of the positive test results turned out to be wrong based on follow-up testing. These expanded tests for rare diseases are performed from a blood draw at 10 weeks of pregnancy and aim to detect chromosomal abnormalities linked to rare diseases. Companies that offer these controversial tests counter that it's only a screening test—not a diagnosis—picking up true positives and noting that patients can and should be counseled about what the findings mean. (Some users may encounter a paywall.)

Somatic expansion, the lifelong growth of specific repeating DNA sequences, is being studied as a potential treatment target for Huntington's disease and other disorders.

See how to make a baby with 3 parents, a process called mitochondrial replacement therapy

This primer video dives into the science of mitochondrial disease risks, how this controversial mitochondrial replacement technique can work, and why proponents say this reproductive advance—already legalized in the UK and Australia—is needed in the United States.