gene therapy

An RNA-editing gene therapy has been developed that switches off the key driver of common eye conditions affecting diabetics and the elderly. It could be an effective alternative to regular injections of medication directly into the eye.

Researchers have developed a gene therapy technology that uses magnetic fields to control specific brain circuits. In addition to Parkinson’s disease, the tech could be used to treat conditions as diverse as depression, obesity, and chronic pain.

A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage, with human trials set to begin within two years.

Engineered mRNA has turned cells into tiny biofactories, producing medications to successfully treat an inflammatory skin condition and two types of cancer, according to a new study. The tech paves the way for therapies in which patients’ bodies make their own drugs.

It feels like back pain comes for many of us at some point in our lives, and it’s tricky to treat. A new gene therapy, which repairs damaged discs and reduces pain, has shown promise in mouse tests.

CRISPR gene-editing has improved the vision of patients with a form of blindness in a Phase 1/2 clinical trial. The results give new hope to patients with the condition, and show that CRISPR could be put to use in humans to treat a range of conditions.

Cow’s milk contains nanoparticles that can be used to deliver RNA therapy orally, say researchers. With such drugs currently only administrable by injection, the discovery opens the door to cheaper, more accessible treatments for a range of diseases.

By silencing the gene responsible for regulating ‘bad’ cholesterol without altering the primary DNA sequence, researchers have unlocked the potential of epigenetic editing to safely and effectively treat not just this but a wide range of diseases.

Researchers have created a single-dose genetic therapy that, in mice, cleared the protein blockages that cause motor neurone disease and frontotemporal dementia, two incurable neurodegenerative diseases that eventually lead to death.

A breakthrough clinical trial using gene therapy has restored hearing to five children born deaf. After six months, the children were able to recognize speech and hold conversations, raising hopes for wider use in the near future.

Researchers have discovered that mice possess a natural form of gene therapy, a non-coding RNA that can sidestep genetic mutations. They were able to engineer a programmable version that might be used to treat genetic diseases in humans.

The FDA has approved the first cell-based gene therapies for treating sickle cell disease, one of which is the first approved therapy to utilize the CRISPR/Cas9 gene editing technology. It signals advancements in gene therapy and regenerative medicine.