黑料吃瓜群网

is leading a gene therapy trial for children with severe immune deficiency disorder that makes them extremely vulnerable to infections.

The trial for children diagnosed with RAG-1 deficient Severe Combined Immunodeficiency (RAG-1 SCID) who are born without infection-fighting immune cells is offered to patients in Europe through the in the Netherlands.

The trial is led by , principal investigator at the and from LUMC鈥檚 Willem-Alexander Children鈥檚 黑料吃瓜群网.

Staal has recently partnered with , a paediatric oncologist and co-lead of the Bone Marrow Transplantation Group from Murdoch Children鈥檚 and The Royal Children鈥檚 黑料吃瓜群网, to broaden a clinical trial site to Australia. Conyers is also an Associate Investigator with the Novo Nordisk Foundation Center for Stem Cell Medicine.

The Murdoch Children鈥檚 trial will see stem cells taken from the child鈥檚 own bone marrow and provided with a healthy copy of the RAG-1 gene in a specialised laboratory. The genetically modified stem cells are then injected into the child鈥檚 bloodstream where they become healthy white blood cells that will build a new, functional immune system.

Under the trial, the stem cells will be transferred to LUMC and treated in a specialised laboratory before being infused back into patients in Australia.

鈥淕iven the patient is their own donor, we avoid the major problems that can come with stem cell transplants,鈥� Conyers said.

鈥淲ith the addition of the Melbourne site, we will reach two important milestones. We make this life-saving therapy available for the first time to Australian babies, but we will also tick over the 10 patient mark of those who have completed our trial to show its efficacy,鈥� Staal said.

The Australian arm of the clinical trial will be led by Murdoch Children鈥檚 and run at The Royal Children鈥檚 黑料吃瓜群网, supported by Novo Nordisk Foundation Center for Stem Cell Medicine. It will run in partnership with the Melbourne Children鈥檚 Trials Centre (MCTC) and is aligned with the Advanced Therapies Initiative.

The trial has been made possible through an investment of DKK6.2 million from the Novo Nordisk Foundation Center for Stem Cell Medicine and in partnership with the team at LUMC.

Image credit: iStock.com/ronnachaipark