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: https://www.bionews.org.uk/page_161449