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

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 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.

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.

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

Originally a bacterial defense system against viruses, CRISPR uses guide RNA and a protein called Cas9 to identify and cut specific DNA sequences. Scientists can repurpose this system to switch off faulty genes, insert new ones to treat genetic diseases, or modify organs grown in other animals so that they appear less foreign to a human immune system.

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.

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.

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.

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.

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.

The Human Genome Project met its goals, but its outcomes were overpromised

The Human Genome Project aimed to sequence the entire human genome, and initial hype suggested it would revolutionize disease treatment. In reality, the project's outcomes were more nuanced, failing to deliver immediate cures despite advancing the understanding of genetics.

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.

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.

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.

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.

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.

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.

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.

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.)

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.

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.

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.)

Peptides account for 31 of 370 new drugs approved by US FDA between 2016 and 2023

Among the approved peptide-based drugs is the first-ever treatment for Rett syndrome, a rare inherited neurologic disorder that leads to a progressive loss of motor skills and language. Peptides are particularly attractive for multiple reasons, including their ability to selectively bind to cellular receptors to either activate or inhibit their function.

In 1984, a dying infant lived for three weeks with a baboon heart

Pediatric-sized hearts are very hard to find. They're about the size of a walnut. When a premature baby girl was born with a fatal heart defect in 1984 her care team transplanted a 7-month-old baboon's heart into the patient. The organ was not genetically modified but still the infant lived for several weeks. This roughly hour-long documentary from the medical institution, features the patient's mother, clinician, and more discussing the controversial procedure and aftermath.

A new AI-assisted reproductive technique is finding 'hidden' sperm cells for those with very low sperm counts

Known as the Star system—for Sperm Track and Recovery—it uses artificial intelligence to help identify and locate "hidden" sperm in men with a rare infertility condition called azoospermia. The condition affects about 1% of men, and these individuals have sperm counts low enough that the individual spermatozoa in their ejaculate may be undetectable. In late 2025, the first baby was born after the child's parents used this assistive technique to locate sperm.

How to consider the ethics of 3-parent IVF, which is illegal in the US

Ethical concerns with this controversial procedure include that a little bit of a child's offspring would be from a third person and that the decisions made with this pregnancy will affect numerous generations, making a permanent change to the gene pool.

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.

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

Experimental hantavirus vaccines and therapies, still under development, have long been funded by the US military.

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

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.

Learn how monoclonal antibodies, which mimic the body's antibodies, can fight diseases like Ebola.

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

Larger animals like giraffes, elephants, and blue whales have developed a unique defense against cancer, a phenomenon known as Peto's Paradox

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

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.

After NASA's Voyager 1 captured Earth as a single pixel as it left the solar system in 1990, astronomer Carl Sagan used the image to reflect on humanity's fragility and the responsibility to protect 'the only world we've ever known.'

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.

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

The syncytiotrophoblast is the outer layer of the placenta and the largest human cell, spanning an area about twice that of a king-sized bedsheet.

The tragic Pulitzer Prize-winning image 'The Vulture and the Little Girl' (1993) generated controversy, raised concerns about ethics in photography, and eventually led to the photographer's death.

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

US housing costs have risen by roughly 175% between 1991 and 2026—but a growing number of Gen Zers are becoming homeowners anyway. In 2025, 4% of homebuyers were Gen Z, up from 3% the year before.

A vending machine run with Anthropic's AI went out of business after it was convinced to give away items for free and sell a video game console.

Prince's sexually explicit 1984 track 'Darling Nikki' attracted the ire of the Parents Music Resource Center, an advocacy group that inspired the Parental Advisory sticker, even though the song ends with a Christian message.

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.

Objects can be 3D printed with light by projecting 2D computerized tomography (CT) scans onto rotating cylinders of resin, which solidify upon exposure.

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.