Even if the well-known endosymbiotic theory is right, and once free-living single-celled organisms evolved to become organelles within larger cells, much remains unexplained about...
Team uses mRNA-loaded carriers to target the genetic instructions of an anti-inflammatory protein in immune cells.
Vision restored after de novo Müller glia-derived genesis of rod photoreceptors in mammalian retinas, pointing to potential treatments for blinding diseases such as macular degeneration.
Spontaneous mutation found in 12 of 14 patients with early infantile epileptic encephalopathy.
A technology designed to improve CRISPR-Cas9 gene editing in mosquitoes and other arthropods succeeds with a high degree of efficiency, while eliminating the need for difficult microinjection of genetic material, according to researchers.
Quethera has developed an ophthalmic gene therapy program designed to treat glaucoma and other conditions affecting the optic nerve, using a rAAV system designed to introduce therapeutic genes into target retinal cells.
Buyer adds Pompe disease candidate, two undisclosed gene therapies in expansion of pipeline of rare neuromuscular disease treatments beyond Duchenne muscular dystrophy.
The selloff was touched off after Spark released second-quarter results in an announcement that included updates on its clinical programs.
Allergan subsidiary exercises option to commercialize CRISPR genome editing treatment candidate EDIT-101 globally for Leber Congenital Amaurosis type 10, a development effort that could generate up to $40 million for Editas.
Collaboration is intended to combine bluebird bio’s expertise in gene transfer and cell therapy with Regeneron’s VelociSuite® platform technologies, with the aim of developing and commercializing new immune cell therapies for cancer.