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.
Exploring ways to prevent birth defects, researchers at the UCL Institute of Child Health have discovered novel supplements that if taken in pregnancy can reduce the chance of the baby having neural tube defects such as spina bifida or anencephaly. These are important in pregnancies where folic acid and other supplements are not as effective. A new patent has been filed we are supporting work to validate and test combinations in animal models.
The blood retinal barrier (BRB), and the blood brain barrier (BBB), represent a significant impediment to drug delivery to the eye and the brain, respectively. This lack of drug penetration has resulted in diseases, such as Age-related Macular Degeneration (AMD), Alzheimer's Disease and Stroke being extremely difficult to address.
Although therapies to eye disease such as AMD already exist, they are delivered by intraocular injections with high risk of side effects related to the administration, the most significant being retinal detachment. The current challenge is to develop drug-delivery systems that ensure transitions across these barriers in a safe and effective manner.
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.
Clinicians at Moorfields Eye Hospital, the leading provider of eye health services in Europe have developed the first medical device which is able to quantify dry eye in a patient. The software quantifies the number, distribution and size of fluorescein staining allowing screening, diagnosis and monitoring of the dry eye and other eye diseases.
AMD is the most common cause of irreversible vision impairment in the Western world and susceptibility to it is influenced by age, environmental and genetic factors. There is currently no cure for AMD and all the efforts concentrate on managing symptoms and early detection and treatment of AMD significantly reduces vision impairment. Most patients are diagnosed at advanced stages of the disease, while genetic testing could assist prediction and medical intervention before the disease progresses. Genetic risk prediction tests are available on the market but the known loci vary in their association with risk making it hard to definitely establish how likely it is for the patient to develop the disease. Researchers at the UCL Institute of Ophthalmology have now identified new sequence variants which can improve the risk prediction accuracy allowing better counseling, helping patients to make the necessary lifestyle changes and therapy before significant vision loss.
During genome transcription, both coding and non-coding transcripts are generated with a wide range of both size and coding potential. Among these are long non-coding RNA (lncRNA) genome transcripts, some of which are antisense to protein-coding genes (AS-lncRNAs). These AS-lncRNAs have been shown to regulate chromatin state, transcription, RNA stability, and translation of the gene.
Researchers at UCL have developed a therapeutic RNA molecule that corresponds with an AS-lncRNA, which can modulate the expression of a target gene. This has potential as a novel therapy for diseases where therapeutic gene suppression is desirable, for example therapeutic reduction of the tau protein in Alzheimer's Disease and other tauopathies.
Epilepsy affects around 60 million people globally and there remains an urgent need for novel treatment methods. Researchers at UCL Institute of Neurology have developed a novel synthetic engineered potassium channel (EKC) as a gene therapy tool to treat epilepsy and other neurological conditions. The inventors have preclinical results demonstrating their engineered potassium channel reduces seizures in a rodent model of epilepsy.
GlucoCEST is a magnetic resonance imaging (MRI) technique that enables visualisation of natural, non-radioactive glucose that can be used in cancer diagnosis. However, this technique has limitations due to interactions with the body glucose sensing system.
To solve this problem, researchers at UCL Institute of Neurology and Department of Chemistry have developed a novel set of imaging agents based on liposome-encapsulated sugar compounds that addresses these issues.