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Personal profile
Academic Biography
I joined Teesside University in July 2022 having been at Royal Holloway University of London for nearly 20 years. After many years in the lab, I became an academic in 2015 having secured a 5 year lectureship fellowship from Muscular Dystrophy UK. My research focuses on the pre-clinical development of gene therapies for rare diseases in particular those affecting the neuromuscular system, including the muscular dystrophies and the childhood cancer neurofibromatosis type 1. The technologies being used include vector-mediated gene addition, endonuclease-mediated gene editing, antisense oligonucleotide (AO)-induced exon skipping and gene silencing.
I interact well with industry and have seen some of my patented therapies enter clinical trial, one of which has received accelerated conditional FDA approval. Future work will concentrate on further pre-clinical development of therapies for other rare diseases with the prospect of future clinical testing through collaboration with existing pharmaceutical partners.
I have been fortunate to be involved in academic-related activities beyond the HE sector to (i) effectively disseminate my research and (ii) contribute to the gene therapy field at large. These activities include:
- Delivering invited presentations at a number of parent/patient conferences (Nov 2013, Oct 2016, Nov 2019)
- Organising patient/parent lab visit days (Jul 2017, Feb 2019, May 2022)
- Being Interviewed by Society of Physiology to provide a lay video on applications of gene editing (Nov 2016)
- Appearing on BBC Radio Surrey to talk about my research (Aug 2015)
- Taking part in Facebook Q&A session organised by a charity on my research (Feb 2018)
- Being invited to present my research on number of occasions at public event on Rare Diseases (Feb 2015, Feb 2020, Feb 2022).
- Being an elected Board member of British Society of Gene and Cell Therapy (BSGCT) sitting on the Public Engagement Sub-Committee that acts to engage and disseminate research findings to general public
- Being an invited member of the External Scientific Committee of Duchenne Parent Project Spain
- Being an invited panel member on the Muscular Dystrophy UK Research Committee since May 2022
- Being invited to make a video for use in teaching A-level biology in schools
- Being interviewed by students from ACS, Egham (March 2018)
- Presenting and participating in an A-Level Science Symposium at Stroudes College, Egham (March 2018)
Affiliations
On the basis of my research, I was invited to join the MDEX Consortium, which is a group of high-profile UK researchers working in the field to allow effective collaboration and exchange of ideas.
Having contributed to the writing of the application to secure the grant, I am part of the Management Committee of a current European Collaboration in Science and Technology (eCOST) Action, I hold responsibility for awarding funds to scientists from Inclusiveness Target Countries (ITC) to facilitate their attendance at international conferences.
Until recently, I was an elected board member of the British Society of Gene and Cell Therapy.
Education/Academic qualification
PhD, Biology, University of Southampton
Award Date: 1 Jul 1993
External positions
Visiting Professor, Royal Holloway University of London
4 Jul 2022 → …
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Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
Bourdon, A., François, V., Zhang, L., Lafoux, A., Fraysse, B., Toumaniantz, G., Larcher, T., Girard, T., Ledevin, M., Lebreton, C., Hivonnait, A., Creismeas, A., Allais, M., Marie, B., Guguin, J., Blouin, V., Remy, S., Anegon, I., Huchet, C., Malerba, A., & 8 others , 2022, In: Gene Therapy.Research output: Contribution to journal › Article › peer-review
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Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy
Lu-Nguyen, N., Malerba, A., Antoni Pineda, M., Dickson, G. & Popplewell, L., 28 Apr 2022, In: Human Gene Therapy.Research output: Contribution to journal › Article › peer-review
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Optimized lentiviral vector to restore full-length dystrophin via a cell-mediated approach in a mouse model of Duchenne muscular dystrophy
Meng, J., Moore, M., Counsell, J., Muntoni, F., Popplewell, L. & Morgan, J., 17 May 2022, In: Molecular Therapy - Methods and Clinical Development. 25, p. 491-507 17 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I
Leier, A., Moore, M., Liu, H., Daniel, M., Hyde, A. M., Messiaen, L., Korf, B. R., Selvakumaran, J., Ciszewski, L., Lambert, L., Foote, J., Wallace, M. R., Kesterson, R. A., Dickson, G., Popplewell, L. & Wallis, D., 14 Jun 2022, In: Molecular Therapy - Nucleic Acids. 28, p. 261-278 18 p.Research output: Contribution to journal › Article › peer-review
Open Access -
Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment
Lu-Nguyen, N., Malerba, A. & Popplewell, L., 25 Feb 2022, Antisense RNA Design, Delivery and Analysis. Arechavala-Gomeza, V. & Garanto, A. (eds.). 1 ed. Humana Press, Vol. 2434. p. 301-313 13 p. (Methods in Molecular Biology).Research output: Chapter in Book/Report/Conference proceeding › Chapter
Open Access