N
Nicholas D. Mazarakis
Researcher at Imperial College London
Publications - 46
Citations - 1947
Nicholas D. Mazarakis is an academic researcher from Imperial College London. The author has contributed to research in topics: Transduction (genetics) & Motor neuron. The author has an hindex of 17, co-authored 46 publications receiving 1672 citations. Previous affiliations of Nicholas D. Mazarakis include Oxford BioMedica & Hammersmith Hospital.
Papers
More filters
Journal ArticleDOI
Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial
Stéphane Palfi,Jean Marc Gurruchaga,G. Scott Ralph,Helene Lepetit,Sonia Lavisse,Philip C. Buttery,Colin Watts,James Miskin,Michelle Kelleher,Sarah Deeley,Hirokazu Iwamuro,Jean Pascal Lefaucheur,Claire Thiriez,Gilles Fénelon,Gilles Fénelon,Cherry Lucas,Pierre Brugières,Inanna Gabriel,Kou Abhay,Xavier Drouot,Naoki Tani,Aurélie Kas,Bijan Ghaleh,Philippe Le Corvoisier,Patrice Dolphin,David P. Breen,Sarah L Mason,Natalie Valle Guzman,Nicholas D. Mazarakis,Pippa A Radcliffe,Richard Harrop,Susan M. Kingsman,Olivier Rascol,Stuart Naylor,Roger A. Barker,Philippe Hantraye,Philippe Remy,Philippe Remy,Pierre Cesaro,Pierre Cesaro,Kyriacos A. Mitrophanous +40 more
TL;DR: ProSavin was safe and well tolerated in patients with advanced Parkinson's disease and a significant improvement in mean UPDRS part III motor scores off medication was recorded in all patients at 6 months and 12 months.
Journal ArticleDOI
Familial amyotrophic lateral sclerosis is associated with a mutation in D-amino acid oxidase
John Mitchell,Praveen Paul,Han-Jou Chen,Alex G. Morris,Miles Payling,Mario Falchi,JJ Habgood,Stefania Panoutsou,Sabine Winkler,Veronica Tisato,Amin Hajitou,Bradley N. Smith,Caroline Vance,Christopher Shaw,Nicholas D. Mazarakis,Jacqueline de Belleroche +15 more
TL;DR: A unique mutation in the D-amino acid oxidase gene (R199W DAO) associated with classical adult onset familial amyotrophic lateral sclerosis (FALS) in a three generational FALS kindred is reported, after candidate gene screening in a 14.52 cM region on chromosome 12q22-23 linked to disease.
Journal ArticleDOI
Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.
Mimoun Azzouz,Thanh Le,G. Scott Ralph,Lucy E. Walmsley,Umrao R. Monani,Debbie C.P. Lee,Fraser J. Wilkes,Kyriacos A. Mitrophanous,Susan M. Kingsman,Arthur H.M. Burghes,Nicholas D. Mazarakis +10 more
TL;DR: It is reported that lentivector expressing human SMN was successfully used to restore SMN protein levels in SMA type 1 fibroblasts and extend survival by SMN expression constructs.
Journal ArticleDOI
Lentivirus-Mediated Gene Transfer to the Central Nervous System: Therapeutic and Research Applications
Liang-Fong Wong,Lucy Goodhead,Christine Prat,Kyriacos A. Mitrophanous,Susan M. Kingsman,Nicholas D. Mazarakis +5 more
TL;DR: The applications of lentiviral vectors in therapy and research are discussed and the essential features that will ensure their translation to the clinic in the near future are highlighted.
Journal ArticleDOI
Dopamine Gene Therapy for Parkinson’s Disease in a Nonhuman Primate Without Associated Dyskinesia
Béchir Jarraya,Sabrina Boulet,G. Scott Ralph,Caroline Jan,Gilles Bonvento,Mimoun Azzouz,James Miskin,Masahiro Shin,Thierry Delzescaux,Xavier Drouot,Anne-Sophie Hérard,Denise M. Day,Emmanuel Brouillet,Susan M. Kingsman,Philippe Hantraye,Kyriacos A. Mitrophanous,Nicholas D. Mazarakis,Stéphane Palfi +17 more
TL;DR: A gene therapy protocol in which the genes that encode the key dopamine biosynthetic enzymes are introduced directly into the brain to produce a perpetual, artificial dopamine factory in neurons of the striatum, the basal ganglia nucleus that receives most of the substantia nigra–released dopamine.