The BSGCT Public and Patient Engagement Subcommittee are organising a series of talks designed to provide a lay introduction and background to the rapidly developing world of gene and cell therapy.
Details will be announced shortly.
What’s Possible with AAV Gene Therapy for Rare Disease
Followed by an “Ask me Anything”
18:30 BST, Wednesday 25th May 2022
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 is generously supported by:
18:30, Wednesday 19th January 2022.
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."