Gene therapy treats a disease by editing its DNA source

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.

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.

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.

Changing a single letter in DNA can permanently alter an organism

Genetic mutations occur when cells replicate, which requires a copy of DNA to be created, and involve substituting, deleting, or inserting letters in a cell's genetic code. Mutations can be caused by normal cellular processes, chemical exposure (e.g., carcinogens), or radiation and may result in genetic disorders or genetic variations that drive evolution.

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.

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.

Genetic engineering has accelerated millennia of altering various crops' genes

Although farmers have long cross-bred crops to obtain plants with specific colors, sizes, and other characteristics, these processes took significant time. Today, genetic modification in agriculture involves scientists altering an organism's DNA for various purposes, including enhancing pathogen resistance, removing allergens, and delaying spoilage.

Recombinant DNA technology turns microorganisms into protein factories

Genes for molecules such as insulin can be inserted into loops of bacterial DNA called plasmids and reinserted into bacteria. Once placed in fermentation tanks, these bacteria use the gene to produce these molecules, which are then harvested and purified for eventual use.

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.

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.

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.

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.

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.

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.

TIGR may supplement CRISPR by providing more refined DNA targeting

The tandem interspaced guide RNA is primarily found in viruses—unlike CRISPR, which is bacterial in origin—and is smaller than CRISPR, making it easier to deliver into cells. The TIGR-Tas system appears to read both sides of the DNA double helix and opens a smaller segment of DNA for repairs, improving the precision of single letter modification.

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.

Genetically modified viruses can help construct battery electrodes

MIT scientists' modifications caused the viruses to attract conductive materials, such as gold ions, which the viruses self-assembled into nanowires that could be used in batteries. These components allow batteries to be thinner and flexible enough to be constructed into new shapes.

Gamma Knife surgery uses incredibly precise, high-energy light to treat patients

A form of brain stereotactic radiosurgery, this non-invasive technology concentrates beams of light onto specific tumors to damage the DNA of malignant cells, killing them. The gamma rays used are in the same part of the electromagnetic spectrum as those produced in nuclear reactions, cosmic ray collisions, and radioactive decay.

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.

Homology-directed repair is the DNA repair method used by cells

Environmental factors like X-ray exposure may break a strand of DNA, but cells can use a sister strand to patch the damage. Scientists exploit this system by introducing custom DNA templates, which cells use instead to make repairs.

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.

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

Monoclonal Antibodies mimic the antibodies produced naturally by the immune system. These molecules target specific parts of the virus, preventing them from entering cells or interfering with their ability to cause harm or infection.

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.

See this 201-level explanation about mitochondria and mitochondrial replacement therapy

This 2022 academic presentation from Stanford University's Dr. Gregory Enns offers a detailed overview about mitochondrial therapy approaches. Enns, a pediatric geneticist, explains how well-functioning mitochondria support the human body.

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

How do stem cells actually work?

Stem cells are unspecialized cells that can develop into various cell types while also making exact replicas of themselves. This amazing ability has made stem cells a candidate to treat a wide variety of diseases and conditions—though the vast majority of potential therapies remain in the research stage.

'Endoevolution' refers to the Darwinian selection happening continually inside us

The term, coined by Roxanne Khamsi in her 2026 book "Beyond Inheritance: Our Ever-Mutating Cells and a New Understanding of Health" describes the idea that evolution does not just occur across generations of humans. Instead, mutated cells continuously compete with healthy ones in our bodies, with implications for our tissues and health. Khamsi says these autocorrections are essentially natural gene therapy. 

Photodynamic therapy is one current treatment for age-related macular degeneration

This laser-based treatment is used on advanced "wet" age-related macular degeneration patients, but it's now used less often than in the past because there are newer injection-based drugs to decrease abnormal blood vessel growth.

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.

An Ebola vaccine was developed during the 2018 west African Ebola outbreak, but the 2026 Ebola outbreak is with a different species of the virus, not the one that the vaccine is approved to treat.

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

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

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

Radioactive elements can introduce software errors because the ionizing radiation they produce can detach electrons from atoms, and computer code relies on the distribution of electrons.

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

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.

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.

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

Elk Cloner, the first computer virus to spread in public, was written as a practical joke by Richard Skrenta, a Pittsburgh high school student.

Children are now three times more likely to say they want to be a YouTuber when they grow up than an astronaut, with some even attending 'YouTube camps' that teach kids how to create content for the platform.

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

Alzheimer's can lead to brain shrinkage, with an average of around 2% of volume reduction per year

Doctors differentiate between a phobia and fear by asking patients whether their fear interferes with their day-to-day activities.

Gerrymandering works through two techniques: cracking opposing voters across districts to dilute their power, and packing them into a few to waste their votes in lopsided majorities.

To reduce wrinkles in clothing, research recommends gentle wash cycles with more water and tumble drying with smaller loads and longer times.

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

After her fellow cult members tried to kill her with an LSD-infused hamburger, one member of the Manson Family, the murderous cult led by Charles Manson, decided to testify against the group's leader.

During the French Revolution, a botanist's letter asking about "dear Rhus" (poison ivy) was intercepted, leading authorities to suspect he was conspiring with the Russians.

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.