Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of ...
In an oral presentation at ASGCT, Editas reported that a single dose of EDIT-401 achieved ≥90 percent mean LDL-C reduction across all dose groups in non-human primates (NHPs). ≥90 percent mean LDL-C ...
In a world first, a research team at the University of Zurich has successfully treated mice carrying an inherited form of ...
What was achieved: Researchers corrected SCN1A mutations in mice using prime or base editing, reducing seizures and improving survival. Why it matters: These are the first demonstrations of directly ...
A tiny salamander that can regrow an entire limb may have just revealed one of the biggest clues yet for future human ...
New preclinical data show PBGENE-DMD drove up to a 3x higher dystrophin protein restoration in skeletal muscle and up to 12x higher in respiratory muscle in early-juvenile mice compared to ...
New POLARIS Phase I/II data presented at ASGCT show ETX101 gene therapy reducing seizures and improving neurodevelopment in ...
Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the closing of $130.0 million aggregate principal ...
Scientists studying axolotls, zebrafish, and mice have uncovered a shared set of genes that may one day help humans regrow ...
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