CRISPR can't fix faulty mitochondria

Despite its potential, CRISPR still fails 15% of the time

Biochemists from UIC found that persistent Cas9 binding to double-strand breaks impacts DNA repair efficiency and the effectiveness of the gene editing tool. RNA polymerase collisions also dislodge Cas9, which affects the efficiency of the tool.

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

3-parent IVF removes most—but not all—faulty mitochondria

The process scoops out about 99% of the mother's mitochondrial DNA—replacing it with that of a donor woman—but because that roughly 1% is still in the system there is still a small chance of disease for the resulting child or future generations. (Some users may encounter a paywall.)

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.

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.

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.)

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.

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.

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.

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.

Without 3-parent IVF, women carrying faulty mitochondrial DNA have few options for children

Women who are carriers of mitochondrial diseases that wish to have children without those conditions cannot be biological parents without three-parent IVF. The alternatives are to adopt or have a child via egg donation.

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.

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.

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.

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.

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.

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.

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.

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.

See how to make a baby with 3 parents, a process called mitochondrial replacement therapy

This primer video dives into the science of mitochondrial disease risks, how this controversial mitochondrial replacement technique can work, and why proponents say this reproductive advance—already legalized in the UK and Australia—is needed in the United States.

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.

How MIT researchers have used genetic mutations to improve photosynthesis efficiency

Recognizing that RuBisCO—a protein that binds carbon dioxide to other compounds to produce the building blocks of organic molecules—often mistakenly binds oxygen, scientists performed directed evolution experiments to modify the geometry of a bacterial version of the protein to cause it to preferentially interact with carbon dioxide over oxygen.

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.

3-parent baby approach may help women with infertility

Though the topline promise of this approach is avoiding passing on mitochondrial diseases, some researchers and companies have been eyeing its potential for helping with female infertility, since faculty mitochondria may contribute to the condition. Recent research suggests this approach could be promising.

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.

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.

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.

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.

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.)

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.

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

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.

Mitochondrial DNA swapping can occur before or after fertilization

Mitochondrial replacement therapy can be conducted with four different techniques, with varying genetic material transferred depending on the approach. Maternal spindle transfer, one common method, involves transplanting the nuclear DNA from a mother into an unfertilized egg from a donor. The donor egg is then fertilized and implanted into the mother's uterus using in vitro fertilization. Another major approach, pronuclear transfer, does the genetic swapping after the mother's egg has already been fertilized. This order of operations may be uncomfortable for some individuals, but the success rate with this technique is higher.

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

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.

Four frozen embryos in the US were created via 3-parent IVF technique

Columbia University researchers created four embryos using mitochondrial replacement therapy for four female patients who didn't want to pass on mitochondrial diseases to their children. Yet US law restricts the researchers from implanting the embryos so they remain frozen. (Some users may encounter a paywall.)

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.

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.

Rodent evidence is mounting that dysfunctional mitochondria may be a cause of Parkinson's

Though researchers have long observed mitochondrial malfunction in Parkinson's patients, it has remained unclear whether faulty mitochondria cause the disease or are casualties of it. Rodent research published in 2025 adds further weight to the idea that it's causative.

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.

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.

Mitochondria—only inherited from mothers in most animals—have their own DNA

Mitochondrial DNA, or mtDNA, typically takes the shape of a circle and contains genes that code for proteins essential to the production of energy-containing molecules called ATP. When mitochondria fuse, the mixing of healthy and damaged mtDNA helps alleviate the effects of mutations that may have occurred during mitochondrial division or environmental interactions.

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.

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.

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

Roughly 1 in every 4,300 individuals in the US has mitochondrial disease

Roughly 1,000 to 4,000 kids are born with these conditions each year in the US though many of the diseases are fatal. Mitochondrial disorders often affect the organs that require the most energy, including the heart, brain, and muscles.

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.

Genetic engineering mice is one idea to reduce Lyme disease transmission

Wild white-footed mice are the main carriers of Lyme. A research team is hoping to genetically engineer mice, using CRISPR, to make the animals immune to Lyme. They're proposing a field trial where they would insert a gene with an antibody to Lyme infection to break the disease transmission cycle on a private island and then try it in Nantucket if that goes well. Simply killing the mice isn't an option because it would upend food webs. Unlike traditional vaccines, this "heritable immunization" would pass from one generation of mice to the next.

Challenges for synthetic biology

Can engineering approaches tame the complexity and unpredictability of living systems? An exploration of five challenges for the field and how they might be resolved.

Mosquitoes are the world's deadliest animal, but gene drives could kill them.