How to purify photoreceptors from retina grown in a dish
9 Sep 2015, 4:54 p.m.
Work recently published in Stem Cells has shown that photoreceptor cells can be grown and purified in the lab before being transplanted into a mature host retina. This research, led by BRC-supported Professor Jane Sowden, suggests that cell replacement th
Identification of genetic mutation linked to complications associated with common birthmarks
18 Mar 2016, 5:42 p.m.
Research led by BRC-supported Dr Veronica Kinsler has found that a subset of a common type of birthmark, which is associated with severe complications, is caused by activating mutations in the genes GNAII and GNAQ. These findings could lead to early ident
Impact of EU Referendum vote on gene and cell therapy in the UK
4 Oct 2016, 1:58 p.m.
In a new editorial published in Human Gene Therapy, leading researchers call for urgent action to ensure continued access to critical funding and ongoing collaborative opportunities with the broader scientific community in the European Union.
Jude Starbeam: communicating research through theatre
31 Jul 2018, 12:30 p.m.
GOSH researchers who specialise in childhood brain injury helped shape a new play that tells the story of a little boy astronaut, Jude Starbeam, who gets lost in space.
Less intensive chemotherapy avoids irreversible side effects in some childhood cancers
18 Dec 2015, 1:24 p.m.
Children with a kidney cancer known as Wilms’ tumour, who are at low risk of relapsing, can have their chemotherapy reduced. This finding comes from a European-wide trial that studied the drug doxorubicin.
London consortium secures £5 million to drive world-class university commercialisation
24 Apr 2018, 12:15 p.m.
The National Institute for Health Research (NIHR) GOSH Biomedical Research Centre (BRC) is part of a new £5 million consortium led by Kings College London which will strengthen London’s status as a world centre of commercialisation in medical technologies
Low myostatin levels linked to lack of success of anti-myostatin drugs for muscle-wasting conditions
30 Nov 2017, 2:04 p.m.
Levels of myostatin, a protein that prevents muscle growth, could influence how well the body responds to anti-myostatin treatments for muscle-wasting conditions such as Duchenne Muscular Dystrophy (DMD). This breakthrough could help predict which patient
Marking Rare Disease Day at GOSH: DNA keyrings and family trees
4 Mar 2020, 1:28 p.m.
For this year's Rare Disease Day on 29th February, we invited children and young people at GOSH to make their own DNA keyring, map out their family tree, learn more about the genetics of rare diseases and even get a 'rare' new look.
Molecular diagnoses of century-old childhood tumours reveals cancer causing mutations
24 May 2017, 2:29 p.m.
A collection of 100-year old tumour samples held at Great Ormond Street Hospital (GOSH) has helped pinpoint the genetic mutations that cause some of the rarest childhood cancers.
New gene therapy shows promise for life-threatening blood disorder X-CGD
9 Mar 2020, 3:29 p.m.
A new gene therapy developed by researchers at Great Ormond Street Hospital (GOSH) has been used to treat patients with a rare disease called X-linked chronic granulomatous disease (X-CGD), as part of an international study.
A new, genetic diagnostic service for ciliopathy disorders has been launched
18 Dec 2015, 1:18 p.m.
In a collaboration between Great Ormond Street Hospital (GOSH) North-East Thames Regional Genetics and the UCL Institute of Child Health, a new genetic diagnostic service for ciliopathy disorders has been launched for service delivery to the NHS.
New genetic test leads to faster, more accurate diagnosis of childhood eye disorders
11 Dec 2017, 11:21 a.m.
A new test to help diagnose and predict a range of serious childhood eye conditions has been developed by researchers at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (ICH).
New sequencing method can diagnose bone abnormalities in fetuses
22 Apr 2018, 5:10 p.m.
Research led by Professor Lyn Chitty has shown that prenatal exome sequencing in fetuses can provide accurate and timely molecular diagnoses for skeletal conditions and inform pregnancy management.
New technique to aid diagnosis of glycosylation disorders present at birth
28 Jun 2016, 2:21 p.m.
A study, supported by the NIHR Great Ormond Street BRC, investigated the use of a 2D-differential gel electrophoresis (DIGE) method that provides a global analysis of the serum glycoproteome (glycomic profiling). 2D-DIGE allows multiple samples to be run
GSK and Orchard Therapeutics today announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for pat
