gene therapy, cell & molecular biology, genetics & genomics

The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.

Researchers treat mice with retinitis pigmentosa using a gene therapy that reduces cone loss.

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

Evolution of Y chromosome; delivering gene with “bionic ears”; diversity of an important cyanobacterium; charting genome-sequencing progress; blockbuster pharma deals

Using lentiviral vectors to replace mutated genes in blood stem cells, scientists successfully treat two rare diseases apparently without causing harmful side effects.

Mice and ferrets are protected from several deadly viruses when genes encoding “broadly neutralizing antibodies” are delivered into their nasal passages.

Exploiting the unique properties of living systems makes synthetic biologists better engineers.