How personalized gene-editing saved a baby's life

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.

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.

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.

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.

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

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.

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

Precision medicine, offering the right treatment at the right time for individuals, integrates genomics, data analytics, and AI to predict risks, personalize treatments, and improve patient outcomes.

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.

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.

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.)

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.

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.

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.

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.

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.

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.)

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

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.

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.)

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.

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.

The promises and perils of using at-home genetic tests for your health

A primer on genetic testing, personalized medicine, and the promise of pharmacogenomics–the use of genetics to predict an individual's response to a drug.

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.

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.

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.

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.

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.

‘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.

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

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.

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.)

One man lived with a gene-edited pig kidney for 271 days, setting a record, before he received a human kidney.

Four frozen embryos in the US were created via 3-parent IVF technique

Columbia University researchers created four embryos using mitochondrial replacement therapy for four female patients who didn't want to pass on mitochondrial diseases to their children. Yet US law restricts the researchers from implanting the embryos so they remain frozen. (Some users may encounter a paywall.)

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

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.

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.

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.

Tumor genetic profiling can lead to targeted treatments

Genetic profiling of tumors—based on a sample from the tumor, a patient's blood, or both—can help clinicians determine if targeted therapies known to block the growth of certain types of tumors would be appropriate for specific 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.

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.

Advanced drugs are closing in on cancer's deadliest mutations

The protein KRAS, mutated in many cancers, was deemed ‘undruggable’. Now scientists are hoping to save lives with a batch of new compounds that target it.

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.

Tech entrepreneur Bryan Johnson's mission to live forever

Johnson has invested millions of dollars into developing a biohacking program he believes will extend his life beyond typical expectations, relying on medical professionals to reduce his "biological age." Most notably, he drew significant public attention with reports of blood-swapping with his teenage son.

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.

mRNA vaccines alter immune response, not DNA, to combat diseases like cancer.

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

The lack of diversity in clinical trials is undermining the future of precision medicine

Medicine works better when the treatments are tailored to fit each individual person’s biology and history. Homogeneous patient selection in clinical trials means treatments and care are being refined based on only a small range of variables, some of which may not apply to you. This 40-minute podcast episode discusses the historical and current reasons for the lack of clinical trial diversity and what this means for the future of personalized medicine.

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.

Genetic tests aren't always actionable, which is one reason genetic counselors are so vital

This podcast interview with a Harvard Medical School professor focused on cancer genetics and prevention discusses the specifics of genetic testing and insights and the challenges of processing the outcomes of the testing and what may be actionable—or not.