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Expert Opinion on Biological Therapy Volume 15, 2015 - Issue 7
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Review

Current developments in gene therapy for amyotrophic lateral sclerosis

Joseph M ScarrottUniversity of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), 385 Glossop Road, Sheffield, S10 2HQ, UK
, BSc (PhD Student) ,
Saúl Herranz-MartínUniversity of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), UK
, PhD (Postdoctoral Research Associate) ,
Aziza R AlrafiahUniversity of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), 385 Glossop Road, Sheffield, S10 2HQ, UK;King Abdulaziz University, Jeddah, Saudi Arabia
, BSc (Student) (Faculty of Applied Medical Sciences) (Student) (Faculty of Applied Medical Sciences) ,
Pamela J ShawUniversity of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), 385 Glossop Road, Sheffield, S10 2HQ, UK
, DBE MBBS MD FRCP FMedSci (Professor of Neurology) &
Mimoun AzzouzUniversity of Sheffield, Sheffield Institute for Translational Neuroscience (SITraN), 385 Glossop Road, Sheffield, S10 2HQ, UK+44 0 114 2222238; [email protected]
, PhD (Chair of Translational Neuroscience)
Pages 935-947 | Published online: 10 May 2015

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Muhibullah Sayed Tora, Orion Paul Keifer$suffix/text()$suffix/text(), Jason John Lamanna & Nicholas M Boulis. (2017) The challenges of developing a gene therapy for amyotrophic lateral sclerosis. Expert Review of Neurotherapeutics 17:4, pages 323-325.
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Stéphane Mathis, Philippe Couratier, Adrien Julian, Jean-Michel Vallat, Philippe Corcia & Gwendal Le Masson. (2017) Management and therapeutic perspectives in amyotrophic lateral sclerosis. Expert Review of Neurotherapeutics 17:3, pages 263-276.
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Xin Fang. (2016) Potential role of gut microbiota and tissue barriers in Parkinson's disease and amyotrophic lateral sclerosis. International Journal of Neuroscience 126:9, pages 771-776.
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Articles from other publishers (25)

Gonçalo J. M. Afonso, Carla Cavaleiro, Jorge Valero, Sandra I. Mota & Elisabete Ferreiro. (2023) Recent Advances in Extracellular Vesicles in Amyotrophic Lateral Sclerosis and Emergent Perspectives. Cells 12:13, pages 1763.
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Ilaria Giovannelli, Adrian Higginbottom, Janine Kirby, Mimoun Azzouz & Pamela J. Shaw. (2022) Prospects for gene replacement therapies in amyotrophic lateral sclerosis. Nature Reviews Neurology 19:1, pages 39-52.
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Namrata Balhara, Mamta Devi, Ajay Balda, Manisha Phour & Arup Giri. (2023) Urine; a new promising biological fluid to act as a non-invasive biomarker for different human diseases. URINE 5, pages 40-52.
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Ton Fang, Goun Je, Peter Pacut, Kiandokht Keyhanian, Jeff Gao & Mehdi Ghasemi. (2022) Gene Therapy in Amyotrophic Lateral Sclerosis. Cells 11:13, pages 2066.
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Maria Zakharova. (2020) Modern approaches in gene therapy of motor neuron diseases. Medicinal Research Reviews 41:5, pages 2634-2655.
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Gayathri R. Ediriweera, Liyu Chen, Justin J. Yerbury, Kristofer J. Thurecht & Kara L. Vine. (2021) Non-Viral Vector-Mediated Gene Therapy for ALS: Challenges and Future Perspectives. Molecular Pharmaceutics 18:6, pages 2142-2160.
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Yeomin Yun & Yoon Ha. (2020) CRISPR/Cas9-Mediated Gene Correction to Understand ALS. International Journal of Molecular Sciences 21:11, pages 3801.
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M. Nizzardo, M. Taiana, F. Rizzo, J. Aguila Benitez, J. Nijssen, I. Allodi, V. Melzi, N. Bresolin, G. P. Comi, E. Hedlund & S. Corti. (2020) Synaptotagmin 13 is neuroprotective across motor neuron diseases. Acta Neuropathologica 139:5, pages 837-853.
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G.Y. Wang, S.L. Rayner, R. Chung, B.Y. Shi & X.J. Liang. (2020) Advances in nanotechnology-based strategies for the treatments of amyotrophic lateral sclerosis. Materials Today Bio 6, pages 100055.
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Vijay Kumar, Tara Kashav & Md. Imtaiyaz Hassan. 2019. Pathology, Prevention and Therapeutics of Neurodegenerative Disease. Pathology, Prevention and Therapeutics of Neurodegenerative Disease 207 224 .
Erik Allen Lykken, Charles Shyng, Reginald James Edwards, Alejandra Rozenberg & Steven James Gray. (2018) Recent progress and considerations for AAV gene therapies targeting the central nervous system. Journal of Neurodevelopmental Disorders 10:1.
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Osama A. Khairoalsindi & Ahmad R. Abuzinadah. (2018) Maximizing the Survival of Amyotrophic Lateral Sclerosis Patients: Current Perspectives. Neurology Research International 2018, pages 1-12.
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Jorge Gomez-Deza & Christopher E. Shaw. 2018. The Molecular and Cellular Basis of Neurodegenerative Diseases. The Molecular and Cellular Basis of Neurodegenerative Diseases 99 115 .
Satish V. Khadilkar, Rakhil S. Yadav & Bhagyadhan A. PatelSatish V. Khadilkar, Rakhil S. Yadav & Bhagyadhan A. Patel. 2018. Neuromuscular Disorders. Neuromuscular Disorders 15 32 .
Andrew P. Tosolini & James N. Sleigh. (2017) Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis. Frontiers in Molecular Neuroscience 10.
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Maria Grazia Biferi, Mathilde Cohen-Tannoudji, Ambra Cappelletto, Benoit Giroux, Marianne Roda, Stéphanie Astord, Thibaut Marais, Corinne Bos, Thomas Voit, Arnaud Ferry & Martine Barkats. (2017) A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model. Molecular Therapy 25:9, pages 2038-2052.
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Liyu Chen, Clare Watson, Marco Morsch, Nicholas J. Cole, Roger S. Chung, Darren N. Saunders, Justin J. Yerbury & Kara L. Vine. (2017) Improving the Delivery of SOD1 Antisense Oligonucleotides to Motor Neurons Using Calcium Phosphate-Lipid Nanoparticles. Frontiers in Neuroscience 11.
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Thanuja Dharmadasa, Robert D Henderson, Paul S Talman, Richard AL Macdonell, Susan Mathers, David W Schultz, Merrillee Needham, Margaret Zoing, Steve Vucic & Matthew C Kiernan. (2017) Motor neurone disease: progress and challenges. Medical Journal of Australia 206:8, pages 357-362.
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Alan Moore, Carolyn A. Young & Dyfrig A. Hughes. (2016) Economic Studies in Motor Neurone Disease: A Systematic Methodological Review. PharmacoEconomics 35:4, pages 397-413.
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Jennifer Roggenbuck, Adam Quick & Stephen J. Kolb. (2016) Genetic testing and genetic counseling for amyotrophic lateral sclerosis: an update for clinicians. Genetics in Medicine 19:3, pages 267-274.
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Thanuja Dharmadasa, José M. Matamala & Matthew C. Kiernan. (2016) Treatment approaches in motor neurone disease. Current Opinion in Neurology 29:5, pages 581-591.
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Xin Fang, Xin Wang, Shaoguo Yang, Fanjing Meng, Xiaolei Wang, Hua Wei & Tingtao Chen. (2016) Evaluation of the Microbial Diversity in Amyotrophic Lateral Sclerosis Using High-Throughput Sequencing. Frontiers in Microbiology 7.
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Sarah Morgan & Richard W. Orrell. (2016) Pathogenesis of amyotrophic lateral sclerosis. British Medical Bulletin 119:1, pages 87-98.
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Lorelei Stoica, Sophia H. Todeasa, Gabriela Toro Cabrera, Johnny S. Salameh, Mai K. ElMallah, Christian Mueller, Robert H. BrownJrJr & Miguel Sena-Esteves. (2016) Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model. Annals of Neurology 79:4, pages 687-700.
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Chrisna J LeVaillant, Anil Sharma, Jill Muhling, Lachlan PG Wheeler, Greg S Cozens, Mats Hellström, Jennifer Rodger & Alan R Harvey. (2016) Significant changes in endogenous retinal gene expression assessed 1 year after a single intraocular injection of AAV-CNTF or AAV-BDNF. Molecular Therapy - Methods & Clinical Development 3, pages 16078.
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