By combining Transformer-based sequence modeling with a novel conditional probability strategy, the approach overcomes ...

Rare diseases, defined by FDA as “a disease or condition that affects less than 200,000 people in the United States” (1), may ...

Scientists have identified a microbe capable of interpreting a single piece of genetic code in two completely different ways, ...

Researchers discover a unique genetic code in Antarctic archaea that encodes a rare amino acid, potentially advancing protein engineering.

KJ Muldoon received a groundbreaking new treatment earlier this year. The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead ...

ABSTRACT: Context: The incidence of gallbladder cancer is quite low in the US, with an estimate (2013) for new cases of less than 10,000. The rarity suggests a possible shared molecular pathology that ...

Researchers uncovered a microbe that treats one stop codon as both a termination signal and an amino-acid instruction, revealing surprising genetic flexibility that may inspire new ways to influence ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene-writing program, inking a deal that lets it in on a prospect for alpha-1 antitrypsin deficiency (AATD) through a $150 million ...

Nov 24 (Reuters) - (This Nov 24 story has been corrected to fix the pricing for Zolgensma to $2.5 million from $2.1 million in paragraph 4) The U.S. Food and Drug Administration has approved Novartis' ...