Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

A new study suggests genetic changes allow horses to produce more energy, while minimizing the toll the energy takes on cells ...

Research using the naturally occurring retinal degeneration 8 (Rd8) mouse model, which carries a nonsense mutation in CRB1, has demonstrated that retinal lesions contain bacterial species ...

Inherited retinal degeneration (IRD) encompasses various disorders characterized by progressive loss of retinal photoreceptor cells, ultimately leading to vision loss and blindness. Among the ...

Data from Eloxx’s proof-of-concept trial in patients with nonsense mutation Alport Syndrome (NMAS) presented in late-breaking presentation at American Society of Nephrology (ASN) Kidney Week New ...

This study, led by Dr. Hyunji Lee, Associate Professor in the Department of Biomedical Sciences at Korea University College of Medicine, Republic of Korea, created a nonsense mutation by changing ...

The beginning to the year has been eventful in the Duchenne muscular dystrophy (DMD) space, with several trial readouts announced at the Muscular Dystrophy Association (MDA) 2025 meeting, which ...

Gianni Castiglione did not set out to study horses. The Vanderbilt University biologist and his colleagues were interested in how different animal species balance the need to produce energy with the ...

Background: Congenital erythropoietic porphyria (CEP, OMIM #263700) is a rare autosomal recessive disease characterized by skin photosensitivity, hypertrichosis, scarring in light-exposed areas, ...