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 therapy involving the transplantation of new photoreceptor cells, isolated from retina grown in a dish, may be a possible treatment for blindness.
Promising findings from a trial for a new stem-cell based therapy for a rare skin condition have been published in the Journal of Investigative Dermatology. The study, which involved intravenous injections of stem-cells, has led to an improvement in the quality of life for the subjects and their carers.
BRC-supported researcher Dr Kevin Mills has been awarded funding from the NIHR to investigate the regenerative ability of the liver following acute liver failure (ALF). If successful, the team hope that their research will lead to a novel therapy that could buy patients enough time to allow their livers to regenerate naturally, avoiding the need for liver transplants
Researchers at GOSH and ICH have been the first team to identify a novel recessive mutation in the transcriptional regulator LHX4 in a family with severe hypopituitarism – a condition that describes the loss of all pituitary hormones.
A drug for Duchenne Muscular Dystrophy (DMD), originally developed by BRC Theme Lead Professor Francesco Muntoni’s Consortium in the UK, has been filed by Sarepta Therapeutics for accelerated approval by the United States Food and Drug Adminstration (FDA).
A new mutation in the protein STAT2 has been identified in patients with mitochondrial disease. These findings could also be beneficial for more common neurodegenerative diseases, including Alzheimer’s, Huntington’s and Parkinson’s diseases.