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.
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.
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.
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.
Collagen is the most widely distributed class of proteins in the human body and the use of collagen-based biomaterials in the field of tissue engineering is extensive. However collagen hydrogels typically have low mechanical strength, and accordingly may not be suitable for use in tendon and other tissue repair grafts without further processing. Whilst there are methods available to increase the strength of collagen, they are not suitable for fabrication of certain tissues as the shape of tissue needs to be “crafted” which leads to structural weakness, layer separation and heterogeneous cell distribution.
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.
We have a first-in-class optimized small-molecule drug candidate ready for IND enabling studies with superior efficacy and activity against pancreatic cancer compared to existing agents, together with a distinct mechanism of action.
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.
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.