Polycystic kidney disease is the most common inherited cause of kidney failure, affecting 1 in 1000 adults and children worldwide. It is characterized by the growth of multiple cysts leading to loss of normal kidney structure and functions and often results in end-stage renal disease. The researchers at UCL Institute of Child Health have demonstrated that treatment with VEGF-C improved disease severity in animal models of PKD.
Voltage-Gated Sodium Channel Knockout Mice: Tools for Developing Therapeutics for the Treatment of Pain2018-12-04T00:03:17+00:00
Scientists at University College London have developed Voltage-Gated Sodium Channel subtype specific conditional knockout mice which provide invaluable tools for the development of therapeutics for the treatment of pain.
We have developed a novel fluorescent assay for the screening of small molecules targeting dengue virus.
We have demonstrated that an abnormality of the protein Complement Factor H Related 5 (CFHR5) leads to a disposition of complement C3 and kidney failure. we have invented an efficient test for this genetic abnormality and have used it to screen a population. We have also invented an ELISA assay to measure the circulating CFHR5 protein levels in humans. We believe this will be useful in diagnosis and assessment of patients with a variety of diseases including SLE, immune complex glomerulonephritis and perhaps age-related macular degeneration. We have also proposed a role for CFHR5 protein (or agents mimicking its action) in the treatment of these diseases and are currently testing this experimentally.
Alzheimer Disease (AD) is the most common neurodegenerative disorder affecting half of all the people above 80 years of age. The available diagnostic methods are expensive and are not available to all patients. An easily detectable biomarker that could be used for diagnosis and monitoring of AD is needed. Researchers at UCL Institute of Ophthalmology demonstrated that measuring drusen deposits in the periphery of the eye can be used as diagnostic method for AD.
Stem Cell Therapy Replacement for Retinal Ganglion Cells: Treatment for Glaucoma and Other Eye Diseases2018-12-04T00:03:17+00:00
Glaucoma is disease in the eye characterised by the progressive loss of retinal ganglion cells. Stem cell based therapeutics provide a method for restoration of neurons damaged by retinal disease. We have identified a population of neural stem cells in the adult human eye that can be isolated from donor retina. Stem cells can differentiate into retinal neurons in vitro and in vivo following transplantation into animal models of retinal degeneration.
DARC: the Diagnosis of Apoptosing Retinal Cells is a new early stage diagnostic method for glaucoma, Alzheimer's and other neurodegenerative diseases. DARC can also be used as an end point marker for the evaluation of neuroprotective drugs.
All lentiviral vectors described to date contain packaging sequences necessary for the viral RNA genome to be assembled into viral particles. These sequences are unnecessarily reverse transcribed into DNA and permanently integrate into target cells. The researchers at UCL Institute of Child Health have developed a lentiviral platform which does not reverse transcribe the packaging sequences into the target cells.
Histamine is a pleiotropic mediator involved in a variety of physiological processes including neurotransmission, endocrine and vascular processes. It also plays a key role in inflammation and binding to one of its four receptors (H1R-H4R). Researchers at UCL Institute of Ophthalmology have shown that targeting H4R receptor in the central nervous system can be beneficial for patients with intraocular retinal inflammatory disease including non-infectious uveitis.
Fibrosis is the formation of excess fibrous connective tissue in response to injury and inflammation. If it happens in response to injury it is also referred to as scarring. Fibrosis is associated with conditions such as cystic fibrosis, cirrhosis, mucous membrane pemphigoid (MMP), pulmonary fibrosis and scleroderma. It is also a consequence of surgery, and especially causes problems after ocular surgery and is a common cosmetic problem. Researchers at UCL Institute of Ophthalmology have found that alcohol dehydrogenase inhibitors can be used in the treatment and prevention of fibrosis.