Nicholas D. Mazarakis

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

G. Ralph, Pippa A. Radcliffe, Denise M. Day et al. · Nature Medicine · 2005 · 499 citations

Neurodegenerative Diseases, Amyotrophic Lateral Sclerosis, Rnai Protects +8

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. Ralph et al. · The Lancet · 2014 · 432 citations

Gene Therapies, Neurodegenerative Diseases, Dose Escalation +11

Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery

Nicholas D. Mazarakis · Human Molecular Genetics · 2001 · 422 citations · Full text

Viral Replication, Viral Pathogenesis, Immunology +16

Stable gene transfer to the nervous system using a non-primate lentiviral vector

K Mitrophanous, Seonwoo Yoon, Jonathan B. Rohll et al. · Gene Therapy · 1999 · 282 citations

Xenotransplantation, Non-primate Lentiviral Vector, Stable Gene Transfer +9

Familial amyotrophic lateral sclerosis is associated with a mutation in D-amino acid oxidase

John Mitchell, Praveen Paul, Han-Jou Chen et al. · Proceedings of the National Academy of Sciences · 2010 · 243 citations · Full text

Neurodegenerative Diseases, Amyotrophic Lateral Sclerosis, Mendelian Disorder +15