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.
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.
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.
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.
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.
A novel solid formulation for the delivery of antibodies and protein therapeutics into the eye. This has several advantages over current methodologies which involve painful intraocular injections.
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.