Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...
Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...
The FDA’s Marty Makary and Vinay Prasad have laid out a path for CRISPR treatments on demand. An expert weighs in on what it ...
Ecuadorian scientists have, for the first time, edited the gene of a fungus that destroys banana plantations. Using an ...
When Mary Shelley publishedFrankenstein; or, The Modern Prometheus in 1818, she wasn’t just writing a gothic horror novel—she ...
Intellia Therapeutics has acknowledged the death of the elderly patient hospitalized last week in its Phase III trial of assessing nexiguran ziclumeran (nex-z) in transthyretin amyloidosis with ...
We provide a protocol for generating human pluripotent stem cell-derived blood-generating heart-forming organoids, a multitissue model encompassing aspects of human cardiac, endothelial and ...
His decoding of the blueprint for life with Francis H.C. Crick made him one of the most important scientists of the 20th ...
ANTLER phase 1 data demonstrate efficacy and durability on par with autologous CAR-T cell therapy and safety allows for ...
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies ...
ELRIG Drug Discovery 2025 recognizes BubbleFect, a lipid-free cell delivery system that utilizes phase separation peptides for non-viral gene therapy delivery.