Gene therapy developed to treat limb-girdle muscular dystrophy – BioNews

Image: Pixabay

A new gene therapy approach has shown promise in a preclinical mouse model for treating limb-girdle muscular dystrophy 2B (LGMD2B), a rare genetic disorder which causes severe muscle weakness.

Scientists at the Children’s National Hospital in Washington DC have developed a new approach that avoids the need for delivering a large gene or giving a large vector dose to target the muscles. Testing their new approach over a 12-week study period, they determined that a single, low-dose injection of a gene therapy vector led to the repair of injured muscle fibres, improved muscle strength and decreased degeneration.

‘Currently, patients with LGDM2B have no gene or drug-based therapies available to them, and we are amongst the few centres developing therapeutic approaches for this disease’, said Dr Jyoti Jaiswal, lead investigator.

Read more at:

Font Resize