“In an early human trial, a single infusion of a new CRISPR gene therapy cut “bad” LDL cholesterol and triglycerides. In four patients, at the highest dose, it reduced LDL by around 50 percent and triglycerides by about 55 percent after two weeks,” notes The Doomslayer.
Reuters reports that
CTX310 works by switching off a gene called ANGPTL3 through a single, two-hour infusion. It was inspired by studies showing people born with an inactive version of the ANGPTL3 gene have a lower lifetime risk of heart disease with no apparent adverse consequences.
Regeneron’s Evkeeza, which treats a rare genetic disorder called homozygous familial hypercholesterolemia, targets the same gene but requires monthly infusions.
CRISPR’s trial of 15 patients aged 31-68, conducted in Australia, New Zealand and the UK, tested five different doses. All participants had high triglycerides, high LDL cholesterol or both and had failed to respond to other treatments.
Among four patients who received the highest dose, triglycerides on average were cut by 55% and LDL by 50% two weeks after treatment. Levels stayed low for at least two months.
A virus is being used to cure deafness in new gene therapy.
“Doctors in Italy have restored a man’s sight using an innovative gene therapy. The treatment involved injecting a viral vector into the man’s eye to deliver a working copy of a missing gene, allowing retinal cells to produce the protein needed for vision,” reports The Doomslayer.
Another form of gene therapy was used to give hearing to a previously deaf toddler. “A British toddler has had her hearing restored after becoming the first person in the world to take part in a pioneering gene therapy trial, in a development that doctors say marks a new era in treating deafness. Opal Sandy was born unable to hear anything due to auditory neuropathy, a condition that disrupts nerve impulses traveling from the inner ear to the brain and can be caused by a faulty gene. But after receiving an infusion containing a working copy of the gene during groundbreaking surgery that took just 16 minutes, the 18-month-old can hear almost perfectly and enjoys playing with toy drums.”
A recently-developed gene therapy blocks the painful hereditary condition angiodema.
