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.

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 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.

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, 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 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.

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

‘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.

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.

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

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.

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.

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.

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.)

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.

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.

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.

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.

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.

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

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.

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.

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

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.

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.

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.)

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.

New eye implant clinical trial recruiting in 2026

University of Southern California researchers have launched a phase 2b clinical trial of a stem cell implant designed to restore vision in people with advanced dry macular degeneration. Patients will be randomized to receive the stem cell implant or a simulated procedure and placebo implant. This type of larger, second-stage clinical trial is designed to confirm efficacy and dosing and to look for side effects. It's one of the stages that precedes the US Food & Drug Administration approval of a device.

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.

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.)

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.

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.

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.)

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

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.

What conditions can be treated with stem cells?

Stem cell research is a global enterprise, and much of the work is being done in Europe. This page gives readers a good sense of the medical conditions stem cell researchers in Europe focus on, and the specific research challenges they address. This page also offers links to in-depth information about many of the research projects.

The spread of fraudulent stem cell treatments

All the coverage of research into promising (but often unproven) medical treatments involving stem cells has created conditions allowing some people to engage in activities regulators describe as scams. As this news article explains, attorneys general in at least seven states have taken legal action against businesses that promised (costly) stem cell treatments but allegedly engaged in fraud.

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.