VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

…, SM Kingsman, P Carmeliet, ND Mazarakis - Nature, 2004 - nature.com
Amyotrophic lateral sclerosis (ALS) causes adult-onset, progressive motor neuron degeneration
in the brain and spinal cord, resulting in paralysis and death three to five years after …

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

…, SM Kingsman, KA Mitrophanous, ND Mazarakis… - Nature medicine, 2005 - nature.com
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease resulting in the
selective death of motor neurons in the brain and spinal cord 1 . Some familial cases of ALS are …

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

…, DP Breen, S Mason, NV Guzman, ND Mazarakis… - The Lancet, 2014 - thelancet.com
Background Parkinson's disease is typically treated with oral dopamine replacement therapies;
however, long-term treatment leads to motor complications and, occasionally, impulse …

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

ND Mazarakis, M Azzouz, JB Rohll… - Human molecular …, 2001 - academic.oup.com
In this report it is demonstrated for the first time that rabies-G envelope of the rabies virus is
sufficient to confer retrograde axonal transport to a heterologous virus/vector. After delivery of …

Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces …

…, AJ Kingsman, ND Mazarakis - Journal of …, 2002 - Soc Neuroscience
Parkinson's disease (PD) is a neurodegenerative disorder characterized by the selective
loss of dopaminergic neurons in the substantia nigra. This loss leads to complete dopamine …

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

…, ND Mazarakis, J de Belleroche - Proceedings of the …, 2010 - National Acad Sciences
We report 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 …

[HTML][HTML] Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy

…, AHM Burghes, ND Mazarakis - The Journal of …, 2004 - Am Soc Clin Investig
Spinal muscular atrophy (SMA) is a frequent recessive autosomal disorder. It is caused by
mutations or deletion of the telomeric copy of the survival motor neuron (SMN) gene, leading …

Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia

…, P Hantraye, KA Mitrophanous, ND Mazarakis… - Science translational …, 2009 - science.org
In Parkinson’s disease, degeneration of specific neurons in the midbrain can cause severe
motor deficits, including tremors and the inability to initiate movement. The standard …

Lentivirus-mediated gene transfer to the central nervous system: therapeutic and research applications

…, SM Kingsman, ND Mazarakis - Human gene …, 2006 - liebertpub.com
The management of disorders of the nervous system remains a medical challenge. The key
goals are to understand disease mechanisms, to validate therapeutic targets, and to develop …

PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer's disease model

…, N Mirzaei, EE Irvine, ND Mazarakis… - Proceedings of the …, 2016 - National Acad Sciences
Current therapies for Alzheimer’s disease (AD) are symptomatic and do not target the
underlying Aβ pathology and other important hallmarks including neuronal loss. PPARγ-coactivator…