Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...

Mucopolysaccharidosis Type II (MPS II) also known as Hunter Syndrome is when the body lacks the enzyme to break down sugar ...

Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

LONDON, Jan 31 (Reuters) - Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care ...

Asiawu Imam says the therapy will make a huge difference A gene-editing therapy for sickle cell disease, with a price tag of £1.65m, is to be offered to patients on the NHS in England.

The one-off gene therapy, known as exagamglogene autotemcel (or ‘exa-cel’), has been approved for use on the NHS in England from today (Friday 31 January) by the National Institute for Health and Care ...

expressed in corn seeds induces an immune response when fed to chickens Faster maturation Coho salmon A type 1 growth hormone gene injected into fertilized fish eggs results in 6.2% retention of ...