Gene therapy has experienced an increasing number of successful human clinical trials, leading to numerous FDA-approved therapies based on recombinant viruses for rare disease, cancer, and other ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

The selection of an appropriate promoter is important to the design and optimisation of adeno-associated viral (AAV) vector-based cardiac gene therapies. The expression cassette design can impact ...

An innovative method that uses modified versions of a bacterial virus effective at delivering treatments to human cells shows promise as a more inexpensive and efficient way to treat some deadly ...

Adeno-associated viruses (AAVs) are common vectors in gene therapy but can frequently cause liver complications in patients. The mechanisms underlying AAV-related liver toxicity remain poorly ...

In a preclinical study, UC Davis Comprehensive Cancer Center scientists developed a highly targeted gene therapy that could revolutionize treatment for cancers linked to a common herpesvirus, with ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Gene therapy achieved its first major breakthroughs in the early 1990s with the treatment of a rare disease called severe combined immunodeficiency. However, the field was dealt a serious setback in ...

The idea behind intra-articular gene therapy for treating osteoarthritis (OA) is to deliver the gene-altering vector or cells directly to the precise site of the disease with a single injection — so ...

A new gene therapy appears to be safe in patients diagnosed with Friedreich ataxia cardiomyopathy, a progressive and fatal inherited cardiac disease, according to a phase 1 clinical trial led by Weill ...

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis. Primarily an inherited disease, its main symptoms concern degeneration of the ...

Muscle spasticity is a common and often debilitating consequence of spinal cord injury that can significantly affect mobility and quality of life. Spasticity is a condition in which muscles become ...