Estrogen-related receptors (ERRs) regulate muscle mitochondria in mice, revealing a new potential drug targets for metabolic diseases.

The scientists at the University of California, San Francisco, and Johns Hopkins University in Baltimore injected an ...

Collectively, the data from in vitro and in vivo studies using FUS, TDP-43, and C9orf72 models demonstrate that hUPF1 holds significant potential for treating the majority of patients with ALS and ...

This multi-center, open-label, dose-escalation clinical trial in the U.S. will assess the safety, tolerability and efficacy of KH658 as a single suprachoroidal space administration of gene therapy for ...

Pacira BioSciences, Inc. announced that it will present findings from a Phase 1 study of its gene therapy candidate, PCRX-201 (enekinragene inzadenovec), for knee osteoarthritis at the 28th Annual ...

A boy with an ultra-rare immune disease which can carry a “death sentence” is living a normal life after taking part in a groundbreaking gene therapy trial. Four-year-old Eisa Hussain now ...

Many diseases are caused by a missing or defective copy of a single gene. For decades, scientists have been working on gene therapy treatments that could cure such diseases by delivering a new copy of ...

Martin, 20, was the first patient in St. Louis and among the first across the country to receive a new gene therapy for sickle cell disease. The monthslong therapy involves extracting a patient ...

This combined approach provides a spatial atlas of gene regulation and defines molecular signatures associated with liver disease for targeted therapeutics or as early diagnostic markers of ...

Caribou was one of the first biotech companies formed to turn CRISPR gene editing into medicines for humans ... The company is now pinning its hopes on CB-010, a therapy for non-Hodgkin’s lymphoma, ...

Intas Pharmaceuticals has announced a series of leadership appointments across several core areas, including gene therapy, information technology, quality control, and learning and development. The ...