How personalized gene-editing saved a baby's life

Gene therapy, a hope of the Human Genome Project, saves a baby's life

A baby with a rare genetic disorder received a bespoke gene-editing treatment using CRISPR technology. The case highlights the potential of personalized medicine for rare diseases, building on the foundation set by the Human Genome Project.

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.

Gene therapy treats a disease by editing its DNA source

This modification is typically performed by viruses, which are naturally skilled at entering cells but have their disease-causing component removed before delivery. Once inside, cells can use the inserted genetic material to produce more helpful proteins, fewer harmful ones, or create new proteins.

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 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.)

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.)

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.

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.

‘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.

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.

One man lived with a gene-edited pig kidney for 271 days, a record

In October 2025, Tim Andrews had his gene-edited pig kidney removed because it was no longer working well. He had the transplanted organ for 271 days—surpassing the prior record of a patient who functioned with one for 130 days. Andrews remains on the kidney transplant wait list to receive a donated kidney from a human, but he has a particularly hard-to-match blood type, making it challenging to get the organ.

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.

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

These vaccines—developed using techniques to create COVID vaccines—are now being tested for cancer treatment and have shown improved survival rates in patients with lung cancer. Scientists believe the research involved in developing these vaccines has opened the door to new and effective personalized cancer treatments in the future.

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.

Mice were able to regenerate retinal cells in the lab—a breakthrough for mammals

Using gene therapy, a team of Korean researchers has enabled retinal regeneration in mice by blocking the intercellular transfer of a single inhibitory protein. This 2025 finding creates a new potential direction for treating retinal diseases. The work, which is still in animal studies, is projected to enter early-stage clinical trials in the next few years.

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.

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.

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.

How two families navigated an unthinkable IVF mistake

The field offers hope to thousands of families annually, but it is also largely unregulated, and for a small number of parents, things can go terribly wrong.

How Gleevec transformed leukemia treatment and cancer research

An outline of the discovery of imatinib (Gleevec), a ground-breaking drug that transformed the treatment of chronic myelogenous leukemia and sparked the beginning of precision medicine.

The Armamentarium is a set of new gene therapy tools to fight brain diseases

Research from 29 universities and institutions across North America has resulted in the development of 1,000 tools that provide large-scale, cell-type specific targeting for the first time. These tools may be used to alter the activity of brain cells to address some neurological diseases and sleep disorders.

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.)

The first prenatal screen for disease was developed in 1961

Scientist Robert Guthrie created a method to test newborns for a metabolic defect called phenylketonuria, or PKU. This blood test didn't look for gene variants—it hunted for excess phenylalanine in the blood. The disorder is caused by an inability to digest the amino acid phenylalanine, leading to excess phenylalanine in the body. PKU results in intellectual disability, seizures, and other symptoms if it isn't caught early and treated with a specialized diet.

How MIT researchers have used genetic mutations to improve photosynthesis efficiency

Recognizing that RuBisCO—a protein that binds carbon dioxide to other compounds to produce the building blocks of organic molecules—often mistakenly binds oxygen, scientists performed directed evolution experiments to modify the geometry of a bacterial version of the protein to cause it to preferentially interact with carbon dioxide over oxygen.

Why don't we all have cancer?

The creation of a single cell produces 120,000 genetic mutations that can lead to cancer. This video investigates how our bodies proofread and autocorrect those mistakes.

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

The first line of 'immortal' human cells, which can divide indefinitely and are used in scientific research, was nonconsensually taken from a Black woman.

About 2,000 US babies are born annually without some portion of their limbs, potentially leading to a lifetime of pricey prosthetics.

Read a deep dive on the 'all-purpose healing' peptide BPC-157.

See the evolution of Ebola vaccine and treatment development.

Hookworm infection, though risky, may help fight certain autoimmune conditions by tamping down the immune system's inflammatory response.

Telehealth demand for GLP-1s for weight loss is booming as consumers seek easier access.

In recent years a child anxiety expert has created and tested a novel phobia and anxiety treatment approach for children, focused on changing how parents respond to the fears.

The hormonal disorder PCOS was renamed PMOS in 2026, a move designed to improve care and research by shifting the focus to the hormonal causes of this condition that affects 1 in 8 women.

Watch how algorithms can be designed to train themselves and self-improve without human input, to the point where the optimized code is too complex for programmers to understand.

Bone marrow (soft tissue found inside certain bones) generates roughly 500 billion new blood cells each day

At the beginning of the 20th century, a group of young men ate standard American food—replete with food additives like borax and formaldehyde—as part of government research to better understand food safety.

As of 2025, the most cited papers in science include a review of graphene, methods to improve image recognition, streamlining electron density calculations, and optimizing protein studies.

See images of medical cases of firework injuries shared on this 'Instagram for doctors.' (Warning: Graphic content)

In 1939, baseball star Lou Gehrig delivered his inspirational 'Luckiest Man' speech after retiring due to the health condition that now bears his name.

Studies suggest when you sleep is as important as how much you get, and syncing with your circadian rhythms may lead to less fatigue and improved long-term health outcomes

A Yale student wrote a profound essay about the consulting industry's influence on elite college students that was published in a book of her writing after she died in a post-graduation car accident.

In 2018, 26-year-old missionary John Allen Chau was killed by tribesmen after attempting to reach and evangelize on India's isolated North Sentinel Island

Venture capital is a form of funding provided to early-stage companies with high growth potential, typically in exchange for an equity (or ownership) stake

Explore an interactive map built from more than 100,000 survey responses on where people find joy in their lives

Massive 3D printers have been used to build homes by stacking layers of 'concrete toothpaste,' a fire-resistant material that effectively insulates against outside noise.

The Brothers Grimm were lawyers before they were storytellers, but became interested in fairy tales as they began investigating how rules and customs had been passed down through cultures.

Brandi Chastain, the star of the 1999 US victory over China in the 1999 Women's World Cup, was a last-second choice to take the penalty kick that sealed the victory.