The first CRISPR-approved treatment was for sickle cell anemia in 2023

As of February 2025, there are more than 150 clinical trials using CRISPR technology

The clinical trials are designed to determine whether experimental CRISPR-related therapies are safe and effective. The majority of trials involve treatments for blood disorders, including beta thalassemia.

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.

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.

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.

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.

‘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.

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.

CAR-T is showing promise for treating lupus—previously thought to be incurable

A small number of lupus patients have remained in complete remission for years after undergoing an experimental CAR-T cell therapy that seems to eliminate enough B cells to fuel a "deep reset" of the immune system. CAR-T therapy is also being used experimentally to treat MS, myositis, and other autoimmune disorders. (Some users may experience a paywall.)

This scientist treated her own cancer

Virologist Beata Halassy treated her own breast cancer by injecting the tumor with lab-grown viruses. Though this self-treatment worked, it has not been approved for widespread use. Read to learn more about her fascinating experience, and how it has led to conversations about safety and ethics.

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.

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.

A snapshot of progress in cancer treatments

The American Association for Cancer Research's "Snapshot of a Year of Progress" offers an overview of significant advancements in cancer research and treatment over the past year. Take a quick look at the rapid pace of scientific discovery and the translation of research findings into clinical practice. In 2024 alone, health regulators approved 15 new anticancer therapies alone.

Researchers are breeding genetically modified mosquitoes to be malaria-resistant

Using CRISPR, a team of international researchers is genetically modifying Anopheles in the lab to be malaria-resistant. The insects would be released into the wild to mate with the wild population, passing down this trait. As of early 2026 they had not yet released the mosquitoes into the wild for field trials. Separate CRISPR work seeks to reduce the Anopheles population altogether.

A new daily MS pill is seeking regulatory approval in 2026

In clinical trials, Roche's fenebrutinib showed promise treating primary progressive MS and doubling relapse-free time in patients with relapsing MS as compared to existing treatment. But some liver damage and two possible drug-related deaths in the trials may make it difficult for the drug to net approval. (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.

US regulators have approved an at-home genetic test looking for BRCA gene mutations

The direct-to-consumer genetic test, approved by US regulators in 2018, examines 3 out of the more than 1,000 known breast cancer-linked genetic mutations. It looks for genetic variations that can increase risk for breast, ovarian, and other cancers. These mutations are most commonly found among Jewish people with Eastern European ancestry.

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.

Carrier screening enables individuals without symptoms to see if they carry a gene for disease

Some conditions are autosomal recessive—meaning a person may carry a gene for that condition, but not have symptoms. Cystic fibrosis, sickle cell disease, and Tay-Sachs disease are examples of autosomal recessive diseases in which a person must inherit two copies of a nonfunctioning gene—one from each parent—to develop disease.

Women's wartime suit features silk dyed with CRISPR-modified bacteria

Anna Dumitriu's bioart piece was created to commemorate the 75th anniversary of the first use of penicillin in a human patient and intends to raise awareness about the rapid development of antibiotic-resistant pathogens by using bacteria that have essentially been transformed into their pre-antibiotic era state.

In 2015, the FDA approved the first 3D-printed prescription tablet

Spritam, an epilepsy medication, had been too large and difficult to swallow, particularly due to its inability to dissolve easily. The new pill was designed with porous layers to enhance its dissolvability, paving the way for future 3D printing of drugs.

Bone marrow transplants were the first successful type of stem cell therapies

Bone marrow contains two types of stem cells: Skeletal (develop into bone, cartilage, and more) and hematopoietic, which create new blood cells. The latter are critical in bone marrow transplants, and give the recipient the ability to create healthy blood cells to fight diseases like leukemia.

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

How mRNA vaccines work

mRNA COVID-19 vaccines were developed faster than any vaccine ever made. Even more enticing, the underlying technology may unlock preventive treatments for a wide-range of diseases.

The US military, concerned about potential deployments worldwide, has long funded experimental hantavirus vaccines and therapies.

Collaborations between the military, biotech, and academic researchers led to an experimental antibody treatment for the Andes hantavirus strain—the one that more readily passes between humans. As of spring 2026, it had only been tested in animals. The US Army has also tested a potential hantavirus vaccine in people. It published early findings in 2023 that seemed promising, eliciting some immune system response. (Some users may encounter a paywall.)

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

Read why one man agreed to get a pig kidney in 2025 to replace his own failing organ

The 68-year-old New Hampshire man told USA Today that a motivator for the experimental surgery was his belief that he was going to die anyway, and other people suffering from kidney disease could gain valuable insights. Yet the surgery bought him time—he lived 271 days with a pig organ and later received a human kidney transplant. Clinical trials began in 2026 to test the pig xenotransplants in more people.

Open brain surgery is not required for BCIs inserted via blood vessel injection

Synchron's brain-computer interface, the Synchron Switch, takes advantage of preexisting medical devices, such as stents and catheters. Cells grow over the device over a 90-day period, incorporating it into the vessel wall and minimizing clots.

Sickle cell trait can put people at elevated risk of heat-related illness

This hereditary blood disorder affects the shape and function of red blood cells. A US military study found that among recruits the incidence rate of exertional heat-related illness was 48% higher among those who had sickle cell trait as compared to those without. Easing into intense exercise and gradually increasing exertional frequency and intensity is recommended.

Leonard Thompson was the first human to receive an insulin injection for diabetes

In January 1922, the 14-year-old was the first person to receive an insulin injection to treat diabetes, just months after insulin was isolated in dogs and used to treat their induced diabetes. Thompson had an allergic response to an apparent impurity in the injection, but his second dose, 12 days later, was a success.

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.

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.

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.

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

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.

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.

After 25 people used virtual reality headsets to practice flying with virtual wings—flapping their arms—their visual cortex responded to the wings almost as if they were real arms, treating them as body parts.

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

SpaceX's S-1 or 'prospectus' revealed many previously unknown details about the company, including its unprofitability despite doing $18.7B in revenue in 2025, exactly how many voting shares Elon Musk will have in the company (roughly 85%), and more.

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

Fast-casual restaurant Chili's is experiencing a resurgence, with its parent company recently reporting a nearly 32% increase in sales, thanks to passionate Gen Z fans on TikTok.

Artificial coral reefs have been 3D printed from nontoxic, biodegradable terracotta clay, which coral polyps have used as a foundation for rebuilding marine ecosystems.

While the first exchange-traded fund (a 'basket' of different assets that can be bought and sold on a stock exchange) in the US only launched in 1993, the global ETF market has grown to more than $10T in the short time since.

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

70 years ago today, city officials in Santa Cruz, California, banned rock 'n' roll to protect its youth from corruption after witnessing a 'provocative' concert the night before.

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.

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.

The first horror movie is believed to be George Méliès' 1986 short 'The Haunted Castle,' which pioneered the use of special effects

The Great Salt Lake's distinct dual coloration results from high-salinity organisms that only live in the northern portion and produce a purple protein.