BSGCT WebsiteMailer Banners (2)_edited.jpg

Public and Patient Engagement Series

The BSGCT Public and Patient Engagement Subcommittee organise this series of talks designed to provide a lay introduction and background to the rapidly developing world of gene and cell therapy.

Next Event

Details to be announced soon.

Wednesday 7th June, 18:30 BST

A novel way of designing gene therapy approaches for neurological diseases

Prof Gabriele Lignani, University College London

Dr Gabriele Lignani is an Associate Professor at UCL Queen Square Institute of Neurology in the Department of Clinical and Experimental Epilepsy. He received his PhD in 2012 in Experimental Neuroscience from the University of Genoa and he did his first postdoc at the Italian Institute of Technology. Then he moved to UCL for his second postdoc and was awarded a Marie-Curie individual fellowship to develop new CRISPR-based editing tools to treat epilepsy. In 2018 he started his own lab as Epilepsy Research UK Emerging Leader to further develop novel gene therapies for epilepsy. He then obtained an MRC New Investigator Award to develop new CRISPR-based technologies and obtained several other discovery and translational grants. He is board member of several committees and Associate editor for Frontiers in Gene Editing and Contributing Editor for Epilepsy Currents; he also leads the Athena Swan initiative for gender balance in his Institute. Recently he has been awarded the Harinarayan Young Scientist Award by ILAE for his research in gene therapy for epilepsy, and the Michael Prize 2023 for the best scientific contribution to progress in the field of experimental epilepsy. The focus of his lab is to develop gene therapy and editing techniques for neurological disorders, to study the role of homeostatic plasticity in epilepsy and understand the basic epileptic mechanisms.

Registration is free for members and non-members

Previous Seminars

A novel way of designing gene therapy approaches for neurological diseases

Speaker: Prof Gabriele Lignani, University College London

Dr Gabriele Lignani is an Associate Professor at UCL Queen Square Institute of Neurology in the Department of Clinical and Experimental Epilepsy. He received his PhD in 2012 in Experimental Neuroscience from the University of Genoa and he did his first postdoc at the Italian Institute of Technology. Then he moved to UCL for his second postdoc and was awarded a Marie-Curie individual fellowship to develop new CRISPR-based editing tools to treat epilepsy. In 2018 he started his own lab as Epilepsy Research UK Emerging Leader to further develop novel gene therapies for epilepsy. He then obtained an MRC New Investigator Award to develop new CRISPR-based technologies and obtained several other discovery and translational grants. He is board member of several committees and Associate editor for Frontiers in Gene Editing and Contributing Editor for Epilepsy Currents; he also leads the Athena Swan initiative for gender balance in his Institute. Recently he has been awarded the Harinarayan Young Scientist Award by ILAE for his research in gene therapy for epilepsy, and the Michael Prize 2023 for the best scientific contribution to progress in the field of experimental epilepsy. The focus of his lab is to develop gene therapy and editing techniques for neurological disorders, to study the role of homeostatic plasticity in epilepsy and understand the basic epileptic mechanisms.

Movement Disorders

Speaker: Dr Joanne Ng, University College London

Dr Joanne Ng is a gene therapy researcher at University College London. She trained as paediatric neurologist with an interest in movement disorders. However the lack of impactful treatments for these patients was disheartening and potential application of gene therapy is highly compelling especially with recent regulatory approval of Upstaza. Dr Ng provides an overview of neurotransmitter related paediatric movement disorders and the application of gene therapy for some of these disorders.

Hard to say and hell to live with. The challenge of living with EB

Speaker: Sharmila Nikapota, Founder and Trustee of Cure EB

Sharmila is the founder of Cure EB (formerly Sohana Research Fund) and Sohana’s mother. She graduated from Cambridge University with a degree in veterinary medicine in 1994 and worked in general practice gaining a certificate in veterinary radiology. She stopped work soon after Sohana was born. Optimism within the research community coupled with chronic underfunding of EB medical research led to the idea for a dedicated research fund. She is the driving force behind the fund, planning events, thinking up fundraising challenges, meeting researchers and speaking at events. With the growth of the fund she plans to concentrate on maximising research progress by building collaborations between researchers, other funding bodies and relationships with biotech to realise the ultimate aim of the fund –to #cureEB. She has three other daughters and a dog.
“Watching Sohana suffer the agonising pain of EB everyday is the best incentive to push forward research to find a treatment for for all”

What’s Possible with AAV Gene Therapy for Rare Disease

Speaker: Dr. Nicole Paulk, University of California, San Francisco

Dr. Nicole Paulk is an Assistant Professor of AAV Gene Therapy in the UCSF Department of Biochemistry & Biophysics in San Francisco. Dr. Paulk has a BS in Medical Microbiology, a PhD in AAV Gene Therapy and Regenerative Medicine from OHSU with Dr. Markus Grompe, and completed her Postdoctoral Fellowship in Human Gene Therapy with Dr. Mark Kay at Stanford University. She is a pioneer in the development of next-generation AAV gene delivery platforms and has engineered payloads for gene repair and gene transfer for numerous rare diseases, utilized directed evolution to evolve capsid serotypes with novel tropisms, and has applied comparative proteomic and epigenomic approaches to interrogate challenges in vector manufacturing. Dr. Paulk’s translational research lab at UCSF develops solutions for the biggest problems in gene therapy: cost, delivery and efficacy, with a focus on treatments for rare diseases and cancer.

This seminar was generously supported by AskBio
https://www.askbio.com

Gene Therapy

Speaker: Dr Simon N Waddington, University College London

"The past two years have witnessed such upheaval that the word “unprecedented” seems inadequate. Hitherto unimaginable occurrences are now accepted with little discussion. One of these events was the successful treatment of more than one tenth of the population of the world with genetic therapy. This was, of course, the use of vaccination, using mRNA or adenovirus, to raise immunity to SARS-CoV2. Aside from this stupendous achievement, we now live in a world where previously untreatable diseases such as Spinal Muscular Atrophy Type I are being cured with single intravenous injections of gene therapy vectors. In this talk, I will discuss some of the technologies underpinning this revolution, I will discuss some of the success stories, and where challenges still lie and where we might see advances being made."