Japanese researchers used a nonviral piggyBac system to genetically modify cynomolgus monkeys, enabling more accurate disease ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.

DelveInsight's "LUXTURNA Market Size, Forecast, and Market Insight Report" highlights the details around LUXTURNA, a one-time gene therapy for the treatment of patients with vision loss due to a ...

Researchers at University of Tsukuba have developed "tomoseqr"—a new software tool that enables easy estimation of the ...

A recent study has shed light on the transcriptional regulatory mechanisms of Penicillium expansum, the fungal culprit behind ...

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II ...

Foghorn Therapeutics advances FHD-909 for SMARCA4 tumors, backed by Eli Lilly partnership. Read here to know why we are ...

Genetic engineering in non-human primates has long been limited by the need for virus-based gene delivery methods. Recently, ...

Genetic engineering in non-human primates has long been limited by the need for virus-based gene delivery methods. Recently, ...

PARP7 is a mono-ADP-ribosyl transferase (mono-ART) which has recently gained attention due to its emerging role as a negative ...

The authors have generated important resources such as a reference dataset of early primate development by utilizing single-cell transcriptomic technology together with induced pluripotent stem cells ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...