In the complex process of producing a gene therapy, bioprocessors depend on assays to monitor the attributes of the product at various stages. Scientists at Thermo Fisher Scientific focus on ...

In a fascinating turn of science and narrative, small mice have assisted scientists to go back in time. By inserting a human-specific gene into their genetic code, scientists potentially opened up ...

Benefits of the gene therapy persisted over several years of follow-up. Presymptomatic children with MLD appeared to have the best outcomes. Severe motor impairment or death was significantly less ...

in Bengaluru and CMC Vellore has resulted in the country's first human gene therapy trial for haemophilia, officials announced on Thursday. Haemophilia is a genetic blood disorder in which blood ...

An Israeli study has identified TRIM63 as a significant genetic contributor to hypertrophic cardiomyopathy (HCM)—the most common hereditary heart disease worldwide. The findings, published in ...

Study provides new evidence that sensory stimulation of gamma-frequency brain rhythm may promote broad-based restorative ...

Removing EPO signaling in mouse liver tumors leads to tumor regression along with sensitivity to immunotherapy.

When used in combination with radiation therapy, more than 60% of the mice survived when compared to the mice that only received radiation. "We included radiation because it is the standard ...

Regeneron’s gene therapy for otoferlin-related hearing loss – a genetic cause of deafness – has restored hearing to normal levels in one profoundly deaf child within just 24 weeks.

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...

Cold' tumors are resistant to common immunotherapies. Researchers have uncovered a master regulator that can be manipulated to prevent tumor growth in mice.