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

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.

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.

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 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.

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.

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.

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.

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.

At companies like Ginkgo Bioworks, researchers are reprogramming cells to act as workshops that can create custom molecules, such as medicines and fertilizers, by stitching together DNA sequences—often in novel combinations—before inserting them into an organism's genome.

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.

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 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.

Genetic testing examines gene variants to help explain disease symptoms. It explores how genetic differences between individuals can be used to understand risks of developing diseases like Huntington's Disease or passing variants linked to disease onto offspring.

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.

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.

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.

Genes are segments of DNA, each composed of a sequence of nitrogenous bases. Within the nucleus, these sequences are copied into a single strand of ribonucleic acid during transcription. This strand leaves the nucleus and is translated by ribosomes into a chain of amino acids that folds into a functional protein.

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.

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.

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.

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.

Purdue University scientists are at work on a new cancer treatment that attacks immune system cells that feed and protect cancer cells rather than attacking them. The immunotherapy process promises to become a universal treatment for solid tumors. Thus far, the treatment has been tested and shown to work on human tumor cells in the laboratory and animal models.

The Synthetic Human Genome Project aims to one day construct human DNA, molecule by molecule, in the hopes of creating disease-resistant cells and synthetic chromosomes for study. However, critics warn it may be misused to create enhanced or modified humans.

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.

These segments of DNA—long molecules made of nucleotides that coil into chromosomes—code instructions for making specific proteins, which correspond to different traits. Approximately 98 to 99% of human DNA is non-coding, with functions beyond protein synthesis.

There are many more genes on Earth today than when microbial life first spawned on the planet about four billion years ago. Many of these newer genes grew from genetic mutations, misprints that slowly drift from the original blueprint via cellular division. If these mutations drift far enough, they can establish a new family of genes, though this process can take well over a million years.

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.

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.

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.

If a tree frog was injected with a DNA mixture containing follistatin, a gene that may influence muscle growth, would it become buff? Josiah Zayner hopes so. By learning how to biohack animals, he hopes people are one step closer to feeling more comfortable "whipping up DIY gene therapy."