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.

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.

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.

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.

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

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

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.

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.

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 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.

Peptides like GLP-1 account for 31 of 370 new drugs approved by the US FDA between 2016 and 2023.

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.

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

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.

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.

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.

Genetic drug matching, called pharmacogenomics, can help avoid dangerous drug reactions

Certain gene variants can affect how quickly or slowly medications are broken down in the body, affecting drug effectiveness and the risk of drugs failing. Such testing is often not covered by insurance companies though research indicates if these tests were performed for more than six drugs across the US population, avoided ailments would likely climb into the hundreds of thousands. (Some users may encounter a paywall.)

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.

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.

Using stem cells for bone marrow transplants

Stem cells hold promise for many future medical applications, but only one type of stem cell is currently approved for use. This page offers a clear description of adult stem cells called hematopoietic stem cells, which create the full range of blood cells and are approved for use in treatments such as bone marrow transplants.

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.

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.

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.

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.

‘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.

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.

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.

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 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.

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.

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.

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.

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

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.

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.)

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 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.

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.