This Week's Science

In this week’s Science, two Italian research groups reported on promising results from gene therapy clinical trials. In both studies, hematopoietic stem cells were removed from patients, then tranduced with lentiviral vectors encoding therapeutic genes and returned to the patients. For three children with the rare lysosomal storage disorder metachromatic leukodystrophy, which is caused by mutations in the ARSA gene, treatment appears to have halted progression of the disease.

Get the full story

This story is free
for registered users

Registering provides access to this and other free content.

Register now.

Already have an account?
Login Now.

Related Posts

In Nature this week: researchers classify pancreatic cancer into four subtypes using sequencing, and more.

Genotyping analysis of measles in Ontario indicates the strain there didn't originate from Disneyland or Europe.

The UK Medical Research Council says the proposed path of a new train line could affect research at the Francis Crick Institute.

The House of Lords in the UK approves a bill to allow mitochondrial donation.