The team notes that different forms of SCN1B gene expression may result in different outcomes for the therapy. However, the proof-of-concept is the first step toward a gene replacement therapy for ...

The outcomes of the new treatment, reported in The Lancet, show that gene therapy at an early age can ... it took his brain time, though, to process what he could now see. Sleep can be difficult ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple ...

Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD).

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ... drug through the development process would require way more ...

Rentschler Biopharma is withdrawing from the field of cell and gene therapy, ceasing operations at its Stevenage, UK site, and increasing its focus on biologics (cell culture-based therapeutic ...

The great hope for gene therapy is that one day it will be out ... offered by a veteran scientist is to optimize early on every process involved in AAV-development technologies.

Our independent appraisal committee has approved the use of gene editing therapy exagamglogene autotemcel (exa-cel) for use in the NHS in England, providing a potential cure for some people with ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...