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 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.

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.

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

Stem cell therapies

Stem cells' special attributes interest researchers developing new therapeutic tools for a variety of health problems. Much of this research is done with support from the National Institutes of Health (NIH). This page offers a snapshot of research programs funded by the NIH and the health problems those programs focus on.

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.

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.

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.

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.

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. (Paywall may apply.)

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.

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.

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.

See how CAR-T cell therapy works to help fight cancer

Short for chimeric antigen receptor T-cell therapy, CAR-T Cell Therapy helps augment the body's own ability to attack cancer cells. It's a cell-based gene therapy that alters the genes in a person's T cells—which are a type of immune cell—to help them beat back cancerous growths.

Why CAR-T therapy has become one of the main focus areas for autoimmune diseases

The therapy, originally targeting cancer, involves taking a patient's own T cells, genetically reprogramming them to recognize and attack a specific target and then infusing them back into patients. For autoimmune diseases, the therapy instead programs them to target immune cells that are causing problems, restoring immune system tolerance.

Brain implants are restoring movement and communication in early human trials

Several companies developing brain-computer interfaces have begun testing on humans with promising results. These implants have helped reanimate paralyzed limbs and enable communication, though ethical concerns remain about data privacy.

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.

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 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.

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

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.

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.

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.

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.

Researchers are investigating if nanoparticle treatments could combat allergies

In rodent work, Northwestern University’s Evan Scott (now at the University of Virginia) and his colleagues have been trying to attack allergies with specially designed nanoparticles—miniscule lab-made objects that can penetrate cells. They stud the nanoparticles with a tiny bit of the allergen specific to the allergy (i.e. cashew protein for cashew allergy) and antibodies that shut down allergic response processes.

Synthetic biology transforms cells into custom DNA foundries

At companies like Ginkgo Bioworks, researchers are reprogramming cells to act as workshops that can create custom molecules, such as medicines and fertilizers, by stitching together DNA sequences—often in novel combinations—before inserting them into an organism's genome.

A new type of immunotherapy focuses on healthy cells hijacked by tumors

Purdue University scientists are at work on a new cancer treatment that attacks immune system cells that feed and protect cancer cells rather than attacking them. The immunotherapy process promises to become a universal treatment for solid tumors. Thus far, the treatment has been tested and shown to work on human tumor cells in the laboratory and animal models.

Neural interfaces offer major medical potential but raise serious ethical concerns

Although brain-computer interfaces can help people recover lost functions and allow direct brain-to-computer communication in the future, surveys have shown little public support for using them to boost abilities like memory or focus in healthy people.

How medical robots could cure cancer

Medical robots are on the verge of being able to be injected into the body, where they will be responsible for semi-autonomous behavior, such as detecting cancer cells and distributing medication. This six-minute video dives into the science of these nanorobots, which are smaller than blood cells. Applications of these nanobots could range from disease discovery and condition monitoring to tissue repair and artificial blood delivery.

The world's first commercial biological computer combines neurons with silicon

Australian start-up Cortical Labs unveiled CL1, a hybrid chip designed to help scientists study brain function in real-time. The 800,000 lab-grown neurons on the chip are kept viable for six months via a built-in life support system.

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.

Teaching immune cells to fight cancer

CAR T-cell therapy is a type of immunotherapy, which involves strengthening the immune system to attack cancer cells. Patient T cells, which manage immune responses and are responsible for killing pathogen-infected cells, are collected and re-engineered to produce surface-level proteins called chimeric antigen receptors. These CARs, now able to bind to specific proteins on cancer cell surfaces, are multiplied into the millions and infused back into the patient.

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 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.

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.)

In 2026, researchers at the University of Minnesota created the world's first synthetic cell—a cell made from non-living chemical components—that can eat, grow, divide, and compete with other cells for resources.

Schizophrenia is being studied with pea-sized 'mini-brains'

Brain organoid research is enabling researchers to study distinct electrical firing patterns associated with schizophrenia. These "mini-brains" that are constructed in the lab from patients' cells could lead to better diagnosis approaches and drug development.

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.)

Minorities are largely underrepresented in medical research, which compromises our understanding of how conditions and treatments may affect various populations, but recent efforts, like the African Ancestry Neuroscience Research Initiative and new genome research studies, are seeking to close that gap.