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New investigational gene therapy shows promise for fatal brain disease

14 May 2020, 10:20 a.m.

Early results of an investigational new therapy used to treat a two-year old has shown promise, reports a Manchester team working with researchers from the UCL Great Ormond Street Institute of Child Health (ICH).

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ICH researcher leads global effort to protect children with cancer during COVID-19 pandemic

21 May 2020, 1:59 p.m.

New international guidance will help frontline staff around the world continue to provide effective care for children and young people with cancer, at a time when these services are under great pressure due to the COVID-19 pandemic.

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Professor Paolo De Coppi elected Fellow of the Academy of Medical Sciences

26 May 2020, 1:45 p.m.

Great Ormond Street Hospital (GOSH) surgeon Professor Paolo De Coppi has been elected to the prestigious Fellowship of the Academy of Medical Sciences.

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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

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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

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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.

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Lab-grown mini-organs could offer treatment hope for children with intestinal failure

7 Sep 2020, 4 p.m.

Pioneering scientists at Great Ormond Street Hospital (GOSH), the UCL Great Ormond Street Institute of Child Health (ICH) and the Francis Crick Institute have grown human intestinal grafts using stem cells from patient tissue that could one day lead to pe

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Researchers grow functioning blood vessels from scratch

9 Sep 2020, 4 p.m.

Human cells ‘sent back in time’ to behave as they did in the womb can be used to grow networks of blood vessels in the laboratory, according to research by an international team including the UCL Great Ormond Street Institute of Child Health (ICH). The fi

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Inflammatory syndrome in children is not caused by a special strain of coronavirus

16 Nov 2020, 1:38 p.m.

The world’s first genetic study of coronavirus from children with inflammatory syndrome PIMS-TS, led by researchers at the UCL Great Ormond Street Institute of Child Health (ICH), suggests characteristics of the human host, rather than of the virus itself

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Promising early results in CAR T-cell trial for common childhood cancer

30 Nov 2020, 6 p.m.

Results from a phase I clinical trial Involving the UCL Great Ormond Street Institute of Child Health (ICH) show that CAR T-cell therapy could achieve activity against neuroblastoma tumours, opening the door to progress this approach towards further clini

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Ancient gene mutation found as the cause of rare hereditary condition for GOSH patient

18 Jan 2021, 5:11 p.m.

UK scientists have found that a 7,000-year-old genetic mutation is responsible for a rare form of hereditary motor neuropathy (HMN).

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GOSH-led metformin trial dramatically reduces seizures for people with Tuberous Sclerosis

2 Feb 2021, 4:28 p.m.

A team of researchers led by UCL Great Ormond Street Institute of Child Health (ICH) and Great Ormond Street Hospital (GOSH) has found that metformin – a drug commonly used to treat Type 2 diabetes – can successfully reduce symptoms associated with …

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New Drug Targets for Childhood Cancer Neuroblastoma Identified

8 Feb 2021, 12:13 p.m.

The largest single cell study to date of the childhood cancer, neuroblastoma, has answered important questions about the genesis of the disease. Researchers from Great Ormond Street Hospital (GOSH), the Wellcome Sanger Institute and their collaborat…

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International Day of Women and Girls in Science 2021

11 Feb 2021, 10:39 a.m.

Carrying out research into the best way to fight children’s illnesses has been one of our main ambitions ever since GOSH opened in 1852. We wanted to take this opportunity of International Day of Women and Girls in Science 2021 to highlight just som…

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GOSH research facilities to play key role in world's first coronavirus Human Challenge study

19 Feb 2021, 5:20 p.m.

Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH) is playing a key role in the world’s first COVID-19 human challenge study, which will get underway shortly following approval from the UK’s clinical trials ethics body.

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Rare Disease-Day 2021

28 Feb 2021, 7 a.m.

Rare Disease Day is an international day celebrated in over 100 countries to raise awareness amongst the general public, the clinical community, and decision-makers about rare diseases and their impact on people affected and their families.

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Global collaborative announced to develop new treatments for paediatric diseases

25 Feb 2021, 7 a.m.

Ahead of Rare Disease Day 2021, GOSH will join forces with three other leading children’s research institutions on three continents to decipher paediatric illnesses, including rare diseases, and find better treatments.

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Uninherited mutations cause spina bifida

19 Feb 2021, 4:53 p.m.

Scientists at the UCL GOS Institute of Child Health reveal that new genetic mutations which occur during embryonic development can cause the severe birth defect spina bifida. Many genes have been implicated in spina bifida, and mutations which are n…

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MRI scans more precisely define and detect some abnormalities in unborn babies

17 Mar 2021, 10:57 a.m.

MRI scanning can more precisely define and detect head, neck, thoracic, abdominal and spinal malformations in unborn babies, finds a large multidisciplinary study led by Evelina London Children’s Hospital, Great Ormond Street Hospital and UCL.

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Cellular benefits of gene therapy seen decades after treatment

16 Mar 2021, 4:54 p.m.

An international collaboration has shown the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells have been cleared by the body.

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