gene therapy

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.

The term ‘personalized medicine’ is being used more and more frequently. But what does it mean? This explainer outlines what personalized medicine is, its advantages and some concerns that have been raised in relation to it.

Up to four million Americans may be enduring this common ailment right now. But what if a microbe found in the gut could relieve it and restore gut health? Scientists believe they've found the genetic key that powers this microbe's motility engines.

Following on from the discovery of programmable DNA-cutting enzymes Fanzors, scientists have identified more than 3,600 Fanzors in a broad set of species. It presents a massive opportunity in the development of new medicines, gene therapy and biotech.

A complete spinal cord injury results, tragically, in total paralysis of all limbs and muscles below the injury site. But now, scientists at EPFL have demonstrated in mice a new gene therapy that can regenerate nerves and restore the ability to walk.

Splitting the gene editor used in traditional CRISPR technology creates a more precise tool with significantly less chance of causing unintended mutations, a new study has found. The novel tool could correct half of the mutations that cause disease.

A high resting heart rate, considered anything above 100 beats per minute, has been linked to an elevated risk of serious cardiovascular conditions such as coronary artery disease and stroke. Knowing how genes impact this is vital for preventative care.

For the first time, researchers have pinpointed two genes – NEK2 and INHBA – that are resistant to chemotherapy for head and neck cancers, and found that by silencing them with existing drugs, treatment could become far more effective.