gene therapy, disease & medicine, genetics & genomics

It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye.

Three studies identify unintended consequences of gene editing in human embryos, including large deletions and reshuffling of DNA.

The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.

The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.

The agency is now evaluating the implications of the corrupted data behind Zolgensma and whether to “take action” against the pharmaceutical company.

At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.

Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”

Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.

A double gene-transfer therapy transformed the non-neuronal cells into rod photoreceptors in the retinas of animal models of congenital blindness.

Preliminary data from Sarepta Therapeutics has exceeded expectations, causing a surge in company stock prices this week.