In this Cell & Gene Therapy TIN interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdoc and non-tenured researchers within the UCL Therapeutic Innovation Networks (TINs), Dr Giulia Massaro highlights her Repurposing TIN Pilot Data Fund awarded project, involving the use of chemokines as a novel target to improve peripheral nerve regeneration. What is the title of your project and what does it involve? I am currently working on a project entitled: ‘Developing a novel gene therapy approach for the treatment of obesity’. This p ..read more

Dr Ahad Rahim is an Associate Professor of Translational Neuroscience and Associate Director of Research at the UCL School of Pharmacy. Dr Rahim’s group works on the development of novel therapies for neurodegenerative diseases and recently at the end of 2019, received an MRC DPFS grant of £654,904 to develop gene therapy for infantile neuroaxonal dystrophy (INAD). Please provide an overview of infantile neuroaxonal dystrophy (INAD) and the need to develop a new therapy. INAD is a devastating inherited neurodegenerative condition that affects children. It’s caused by mutations in a gene called ..read more

In this interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdocs and non-tenured researchers at University College London (UCL), Dr Annalucia Darbey and Dr Charlotte Spicer highlight their joint Cell and Gene Therapy Therapeutic Innovation Network (TIN) Pilot Data Fund awarded project, investigating the comparison of novel gene therapies to treat an inherited neurodegenerative disorder.  What is the title of your project and what does it involve? Our project is entitled “Comparing novel AAV gene therapies for the treatment ..read more

In this interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdocs and non-tenured researchers at University College London (UCL), Dr Amy Richardson highlights her Cell and Gene Therapy Therapeutic Innovation Network (TIN) Pilot Data Fund awarded project based on testing a novel gene therapy for epilepsy.  What is the title of your project and what does it involve? My project, ‘Ion transporter gene therapy for epilepsy’, is based on testing a novel gene therapy as a treatment for epilepsy and validating a viral construct in t ..read more

In this Cell & Gene Therapy TIN interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdoc and non-tenured researchers within the UCL Therapeutic Innovation Networks (TINs), Dr Marion Mercier highlights her Cell & Gene Therapy TIN Pilot Data Fund awarded project, involving the validation of novel gene therapies for epilepsy. What is the title of your project and what does it involve? Human brain tissue is routinely excised during epilepsy surgery, and can, given the right conditions, be maintained alive in slice culture for ..read more

In this Cell & Gene Therapy TIN interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdoc and non-tenured researchers within the UCL Therapeutic Innovation Networks (TINs), Dr Rajeev Rai highlights his Cell & Gene Therapy TIN Pilot Data Fund awarded project, involving hematopoietic stem cell gene editing to correct platelet defects in Wiskott Aldrich Syndrome (WAS). What does your Cell & Gene Therapy TIN project involve? Wiskott Aldrich Syndrome (WAS) is an X-linked recessive primary immunodeficiency disease characteri ..read more

In this Cell & Gene Therapy TIN interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdoc and non-tenured researchers within the UCL Therapeutic Innovation Networks (TINs), Dr Juan Antinao Diaz highlights his Cell & Gene Therapy TIN Pilot Data Fund awarded project, involving AAV delivery in Dravet syndrome. What is the title of your project and what does it involve? This project is called “AAV delivery of an NaV1.1 activator for the treatment of a Dravet Syndrome mouse model”. Dravet syndrome is a childhood epilepsy, cause ..read more

In this Cell & Gene Therapy TIN interview as part of the Early Career Innovators series, recognising the amazing translational work being done by postdoc and non-tenured researchers within the UCL Therapeutic Innovation Networks (TINs), Dr Ellie Crompton highlights her Cell & Gene Therapy TIN Pilot Data Fund awarded project, involving new therapies for rare disease Maple Syrup Urine Disease (MSUD).  What is the title of your project and what does it involve? My project is entitled “Development of novel therapies for Maple Syrup Urine Disease (MSUD)”. MSUD is a rare, paediatric, me ..read more

Written by Linda von Nerée, NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at UCL. How many pairs of jeans do you have in your wardrobe? How many genes are in your body? What are genes anyway and do you know how they can help to treat an illness?! All is explained in this brand-new animation from us at the NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at University College London (UCL BTRU). Well, except how many jeans you own, that stays your secret. Young people asking questions about gene therapy in the animation ‘Gene Therapy explaine ..read more