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

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.

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 allows scientists to cut out and replace faulty or unwanted DNA, which can help treat genetic disorders, such as sickle cell disease, and create genetically modified organisms, such as allergen-free foods.

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, has successfully treated a baby born with a rare urea cycle disorder that causes toxic ammonia level buildup.

See this list of FDA-approved gene therapies, including offerings for inherited conditions as well as some forms of cancer.

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

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.

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.

CRISPR turns 10 years old

When Dr. Jennifer Doudna and colleagues released a paper in 2012 demonstrating the ability to harness the cell's natural immunity machinery to edit DNA, it received little fanfare. A decade later, the gene editing technology has led to innovations in medicine, evolution, and agriculture—and raised profound ethical questions about altering human DNA.

Early research suggests CAR-T therapy might treat scleroderma, reducing skin thickening

Clinical data from a couple patients in 2025 indicated that one experimental CAR-T therapy called rese-cel was able to genetically reprogram T cells to eliminate certain problematic immune B cells that were causing the condition. The therapy reduced skin thickening and allowed the patients to halt using immunosuppressive medications and steroids.

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.

Explaining CRISPR at five levels of technical detail

CRISPR is a new area of biomedical science that enables gene editing and could be the key to eventually curing diseases like autism or cancer. WIRED challenges biologist Neville Sanjana to explain this concept to 5 different people; a 7-year-old, a 14-year-old, a college student, a grad student and a CRISPR expert.

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.

CRISPR-Cas9 enables precise genome editing using a simple RNA-guided system

Jennifer Doudna's research uncovered how bacteria use the tool as an immune defense by cutting viral DNA. She and Emmanuelle Charpentier received the 2020 Nobel Prize in chemistry for showing how this mechanism could be engineered to target and edit genes in living cells.

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.)

Advances in agriculture are being powered by CRISPR to boost carbon capture

Researchers are using CRISPR to test thousands of plant gene edits, aiming to speed up breeding and maximize carbon retention in soil. By engineering drought tolerance, they also aim to increase crop yields and expand the environments where crops can grow.

A physician with a rare and deadly disease saved his own life by identifying an existing medication that he repurposed for his condition, and now he's using AI to try to do the same for other patients.

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.

CRISPR can't fix faulty mitochondria

Gene editing via CRISPR could theoretically fix many issues in biology, but editing the mitochondrial genome has remained challenging. In recent years, there has been some progress reaching these cellular powerhouses. (Some users may experience a paywall.)

Drs. Jennifer Doudna and Emmanuelle Charpentier discuss CRISPR

Jennifer Doudna and Emmanuelle Charpentier's were awarded the 2020 Nobel Prize for research focused on understanding and adapting the CRISPR-Cas9 system to precisely edit DNA sequences in other organisms. Doudna has indicated that the system can potentially treat genetic forms of blindness and high cholesterol.

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

Epigenetic editing—altering gene expression by removing or adding chemical groups on DNA—is making significant strides in the lab, even lowering cholesterol in monkey experiments by about 70%. (Free w/email.)

A deeper (nontechnical) dive into how CRISPR works

Extra crispy? No, it's CRISPR, but this gene-editing technology is "extra" in the sense of being momentous and destined to change everything. Through CRISPR, scientist exploit a quirk in bacteria immune systems, allowing them to potentially, and quickly, control genes, remove undesirable traits, and add preferred ones. The possibilities and applications are endless.

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.

A scientist successfully treated her own breast cancer by injecting the tumor with lab-grown viruses.

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.

The 'Armamentarium' is a set of new gene therapy tools—including viruses that could shuttle therapies into the body—designed to fight brain diseases.

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.

Researchers are breeding genetically modified mosquitoes to be malaria-resistant.

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.

Scientists create the first CRISPR-modified spider to enhance its silk proteins

A research group at the University of Bayreuth used CRISPR-Cas9 to genetically modify spiders to produce glowing red silk. Spider silk is five times stronger than steel by weight, and this research paves the way for developing new silk functionalities.

CRISPR technology is removing allergens from common foods

Proteins responsible for allergies to eggs, milk, wheat, peanuts, and other foods can be genetically edited out of foods to reduce the risk of life-threatening reactions. The technique has also been used to produce caffeine-free coffee beans, spicier tomatoes, allergen-free pets, and more nutritious fish.

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.

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.

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

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.

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.)

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.

Malaria mosquitoes in a lab were wiped out using CRISPR

Scientists at Imperial College London disabled a gene in female Anopheles gambiae mosquitoes to make them infertile and propagated the infertility mutation at unnaturally high rates using a gene drive. The entire population collapsed within a dozen generations, showing promise for subsequent with mosquitoes in mimicked tropical environments.

Chinese scientists transplanted a pig lung into a human being in 2025—but it had to be removed.

A clinical trial is underway in 2026 to help kidney transplant recipients avoid a lifetime of immunosuppressing drugs

This later-stage clinical trial, in Chicago and Taiwan, evaluates the efficacy and safety of a new cellular therapy that may be swapped in for immunosuppressing drugs among kidney transplant recipients. The study randomizes people to either receive standard immunosuppressants or this cellular therapy and fewer immunosuppressants. It only includes kidney transplant recipients receiving organs from live donors so transplant surgery timing won't hinge on the unpredictable schedule of performing surgery and follow-up care when a donor dies.

Different enzymes can be used in the CRISPR system to modify how DNA is cut

The system uses RNA sequences to guide enzymes like Cas9 to specific spots in DNA, where they cut or modify genes. Unlike Cas9, the Cpf1 enzyme makes staggered cuts, which helps insert new DNA more cleanly.

Australia legalized 3-parent IVF in 2022

It was the second country, after the UK, to legalize the procedure, which is still prohibited in the US. There are more than 300 rare mitochondrial diseases that can be passed from a mother to her offspring.

Stem cell therapy has shown promise in treating spinal injuries in an early-stage clinical trial.

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.)

Gene therapy for a rare type of deafness is allowing deaf children and adults to hear for the first time, according to research results reported in April 2026.