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Biomedical Research Centre news

First targeted gene therapy for Wiskott-Aldrich Syndrome shows promise

12 Aug 2020, 10:02 a.m.

GOSH researchers have shown the power of gene editing technology CRISPR-Cas9 in correcting DNA ‘spelling mistakes’ that cause rare immune system conditions in children.

Fresh perspectives from children and young people

29 Jul 2020, 4:35 p.m.

Talking to our young patients is so important. They tell us what works, what doesn’t, and how to improve what we do. Recently, we called on the expertise of our amazing patients to develop a series of videos about uveitis – a condition that causes inflamm

Bringing together the UK rare disease community

13 Jul 2020, 1:54 p.m.

Diagnosis with a rare disease - one that affects less than 1 in 2,000 people - can leave a family feeling isolated and alone. But we can support those families and make real progress by bringing the rare disease community together. In December 2019, the N

ICH researchers discover the cause of a new rare genetic syndrome

6 Jul 2020, 3:58 p.m.

ICH researchers discover the cause of a new rare genetic syndrome that causes life-threatening symptoms in children.

ICH researchers develop a technique to 'print' healthy new tissue

24 Jun 2020, 12:51 p.m.

A pioneering international study led by the UCL Great Ormond Street Institute of Child Health (ICH) has seen researchers develop a photosensitive bio-gel that uses light treatment to ‘print’ healthy new tissue directly into specific tissues and organs, an