Professor Linda Popplewell - 'The development of gene therapies for rare diseases'
1:00pm - 2:00pm /
Thursday 9th January 2025
/ Venue: Physiology seminar room Nuffield Wing
Type: Seminar /
Category: Research
/ Series: Institute of Systems, Molecular and Integrative Biology
- Dr Pradeep Harish
- Admission: Free, no registration needed.
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Linda Popplewell has recently joined the National Horizons Centre, Teesside University as Professor of Genetic Medicine. With funding from various bodies, including the Gilbert Family Foundation, LifeArc and industrial partners, she leads a large research group focused on the pre-clinical development of gene therapies for neurofibromatosis type 1 (NF1) and neuromuscular diseases, in particular muscular dystrophies.
The work is focused in the following areas:
(1) Development of antisense oligonucleotides (AOs) to skip mutated exons and restore neurofibromin expression for NF1;
(2) Optimisation of AOs for the skipping of out-of-frame exons to restore dystrophin expression for Duchenne muscular dystrophy (DMD);
(3) Optimisation and in vivo testing of AOs to knockdown toxic DUX4 expression in models of facioscapulohumeral muscular dystrophy (FSHD); (4) Targeting of specific genes with AOs and catalytically-inactive CRISPR/Cas9 tethered to transcriptional activators and inhibitors to modulate the muscle fibrosis and myogenesis;
(5) Development of endonucleases for editing of the dystrophin gene that would have high patient applicability;
(6) Optimisation of AAV vector construct for DMD and oculopharyngeal muscular dystrophy (OPMD), and non-viral vectors for NF1.
She is co-inventor on a number of patents, which have been successfully commercialised through license agreements with various pharmaceutical companies. One of her patented AO designs for DMD has recently been FDA approved.
The work is focused in the following areas:
(1) Development of antisense oligonucleotides (AOs) to skip mutated exons and restore neurofibromin expression for NF1;
(2) Optimisation of AOs for the skipping of out-of-frame exons to restore dystrophin expression for Duchenne muscular dystrophy (DMD);
(3) Optimisation and in vivo testing of AOs to knockdown toxic DUX4 expression in models of facioscapulohumeral muscular dystrophy (FSHD); (4) Targeting of specific genes with AOs and catalytically-inactive CRISPR/Cas9 tethered to transcriptional activators and inhibitors to modulate the muscle fibrosis and myogenesis;
(5) Development of endonucleases for editing of the dystrophin gene that would have high patient applicability;
(6) Optimisation of AAV vector construct for DMD and oculopharyngeal muscular dystrophy (OPMD), and non-viral vectors for NF1.
She is co-inventor on a number of patents, which have been successfully commercialised through license agreements with various pharmaceutical companies. One of her patented AO designs for DMD has recently been FDA approved.