Precision Medicine


Precision Therapies

Targeted therapy - the foundation of precision medicine.

The UCL Institute for Precision Medicine aims to accelerate the development of novel drugs and targeted therapies. Our leading research groups are described here.

Antisense Oligonucleotide Therapies

Developmental Neurosciences
Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy are neurodegenerative genetic diseases characterized primarily by muscle weakness and wasting. Until recently there were no effective therapies for these conditions, but antisense oligonucleotides, a new class of synthetic single stranded molecules of nucleic acids, have demonstrated promising experimental results.

Cell Therapy/Immunotherapy

UCL Institute of Ophthalmology Gene and Cell Therapy Group 
The UCL Institute of Ophthalmology Gene and Cell Therapy Group is developing gene and cell therapy for sight loss by translating basic research into new treatments.

Stem Cell Therapy
The London Project to Cure Blindness was established eight years ago with the aim of curing vision loss in patients with wet age-related macular degeneration (AMD).

UCL Institute of Immunity and Transplantation
The UCL Institute of Immunity and Transplantation (IIT) aims to develop vaccinations, gene therapy and cell therapy to enhance immunity in patients with cancer and chronic infection, and to induce tolerance to autoimmune diseases in patients receiving conventional or bio-engineered transplants.

Gene Therapy                       

UCL Molecular and Cellular Immunology
The Molecular and Cellular Immunology Section at the UCL Great Ormond Street Institute of Child Health researches the molecular basis of primary immunodeficiency disorders, and other related haematopoietic disorders and is developing improved forms of treatment.

UCL Translational Immunotherapy Research Group
The UCL Translational Immunotherapy Research Group studies the immunology of bone marrow transplantation, incorporating immunotherapy, immune reconstitution and graft-versus-host disease (GvHD), with a special focus on translational research.

UCL Prenatal Cell and Gene Therapy
Dr Anna David leads a team at the UCL Elizabeth Garrett Anderson Institute for Women's Health. The team aims to develop prenatal treatment of severe early onset and life-threatening disorders using gene and cellular therapy.

Gene Therapy for Metabolic Diseases
Development of gene therapy in several childhood inherited metabolic disorders.

Neurotransmitter Gene Therapy
Dr Manju Kurian, Consultant Paediatric Neurologist, is looking at a possible gene therapy approach to childhood Parkinsonism.