Therapeutic rAAVrh 10 Mediated SOD 1 Silencing in Adult SOD 1 ( G 93 A ) Mice and Nonhuman Primates

[1]  B. Byrne,et al.  Stimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines. , 2015, American journal of respiratory cell and molecular biology.

[2]  P. Mitra,et al.  Brain-mapping projects using the common marmoset , 2015, Neuroscience Research.

[3]  M. Souweidane,et al.  Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates. , 2015, Human gene therapy. Clinical development.

[4]  J. Mendell,et al.  Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates. , 2015, Molecular therapy : the journal of the American Society of Gene Therapy.

[5]  P. Aebischer,et al.  SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice , 2015, Annals of clinical and translational neurology.

[6]  M. Chillón,et al.  AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy , 2014, Gene Therapy.

[7]  C. Svendsen,et al.  Delayed Disease Onset and Extended Survival in the SOD1G93A Rat Model of Amyotrophic Lateral Sclerosis after Suppression of Mutant SOD1 in the Motor Cortex , 2014, The Journal of Neuroscience.

[8]  B. Byrne,et al.  Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. , 2014, Molecular therapy : the journal of the American Society of Gene Therapy.

[9]  Mackenzie W. Mathis,et al.  Necroptosis Drives Motor Neuron Death in Models of Both Sporadic and Familial ALS , 2014, Neuron.

[10]  Robert H. Brown,et al.  Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. , 2014, Human molecular genetics.

[11]  L. Ferraiuolo,et al.  Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[12]  J. Julien,et al.  Mitochondrial damage revealed by immunoselection for ALS-linked misfolded SOD1 , 2013, Molecular Neurodegeneration.

[13]  A. Walker,et al.  Extracellular wildtype and mutant SOD1 induces ER–Golgi pathology characteristic of amyotrophic lateral sclerosis in neuronal cells , 2013, Cellular and Molecular Life Sciences.

[14]  A. Horwich,et al.  Molecular chaperone Hsp110 rescues a vesicle transport defect produced by an ALS-associated mutant SOD1 protein in squid axoplasm , 2013, Proceedings of the National Academy of Sciences.

[15]  R. Samulski,et al.  Global CNS Gene Delivery and Evasion of Anti-AAV Neutralizing Antibodies by Intrathecal AAV Administration in Non-Human Primates , 2012, Gene Therapy.

[16]  B. van Zundert,et al.  Early pathogenesis in the adult‐onset neurodegenerative disease amyotrophic lateral sclerosis , 2012, Journal of cellular biochemistry.

[17]  Robert H. Brown,et al.  A high-throughput screen to identify inhibitors of SOD1 transcription. , 2012, Frontiers in bioscience.

[18]  K. Foust,et al.  Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. , 2012, Human gene therapy.

[19]  H. Petry,et al.  In vivo knock-down of multidrug resistance transporters ABCC1 and ABCC2 by AAV-delivered shRNAs and by artificial miRNAs , 2011, Journal of RNAi and gene silencing : an international journal of RNA and gene targeting research.

[20]  K. Bankiewicz,et al.  An alpha-synuclein AAV gene silencing vector ameliorates a behavioral deficit in a rat model of Parkinson's disease, but displays toxicity in dopamine neurons , 2011, Brain Research.

[21]  Christian Münch,et al.  Prion-like propagation of mutant superoxide dismutase-1 misfolding in neuronal cells , 2011, Proceedings of the National Academy of Sciences.

[22]  M. Ehrlich,et al.  Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design , 2011, Gene Therapy.

[23]  Jeffery N Agar,et al.  Wild-type and mutant SOD1 share an aberrant conformation and a common pathogenic pathway in ALS , 2010, Nature Neuroscience.

[24]  S. Niclou,et al.  Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system , 2010, BMC Neuroscience.

[25]  Robert H. Brown,et al.  XBP-1 deficiency in the nervous system protects against amyotrophic lateral sclerosis by increasing autophagy. , 2009, Genes & development.

[26]  P. Aebischer,et al.  Dose optimization for long-term rAAV-mediated RNA interference in the nigrostriatal projection neurons. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[27]  E. Beghi,et al.  Incidence of amyotrophic lateral sclerosis in Europe , 2009, Journal of Neurology, Neurosurgery & Psychiatry.

[28]  D. Borchelt,et al.  Variation in aggregation propensities among ALS-associated variants of SOD1: Correlation to human disease , 2009, Human molecular genetics.

[29]  H. Okano,et al.  Generation of transgenic non-human primates with germline transmission , 2009, Nature.

[30]  Takanori Yokota,et al.  Als-linked Mutant Sod1 Induces Er Stress-and Ask1-dependent Motor Neuron Death by Targeting Derlin-1 -induced Cell Death Remains Controversial. Here We Show That Sod1 Mut Specifically Interacted with Derlin-1, a Component of Endoplasmic Reticulum (er)-associated Degradation (erad) Machinery and Trig , 2022 .

[31]  Brian L. Gilmore,et al.  Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi , 2008, Proceedings of the National Academy of Sciences.

[32]  Tetsuya Suhara,et al.  Neurobehavioral protection by single dose l-deprenyl against MPTP-induced parkinsonism in common marmosets , 2007, Psychopharmacology.

[33]  C. Tankersley,et al.  Respiratory impairment in a mouse model of amyotrophic lateral sclerosis. , 2007, Journal of applied physiology.

[34]  Zuoshang Xu,et al.  Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo , 2006, Neurobiology of Disease.

[35]  B. Monia,et al.  Antisense oligonucleotide therapy for neurodegenerative disease. , 2006, The Journal of clinical investigation.

[36]  Theresa A. Storm,et al.  Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways , 2006, Nature.

[37]  L. Greensmith,et al.  Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model , 2005, Nature Medicine.

[38]  C. Henderson,et al.  Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS , 2005, Nature Medicine.

[39]  Robert H. Brown,et al.  RNA interference-mediated silencing of mutant superoxide dismutase rescues cyclosporin A-induced death in cultured neuroblastoma cells. , 2004, Proceedings of the National Academy of Sciences of the United States of America.

[40]  K. Mansfield,et al.  Marmoset models commonly used in biomedical research. , 2003, Comparative medicine.

[41]  G. Filippini,et al.  Treatment of restless legs syndrome with gabapentin: A double-blind, cross-over study , 2002, Neurology.

[42]  Thomas D. Schmittgen,et al.  Analysis of relative gene expression data using real-time quantitative PCR and the 2(-Delta Delta C(T)) Method. , 2001, Methods.

[43]  R. Ridley,et al.  Functional integration of striatal allografts in a primate model of Huntington's disease , 1998, Nature Medicine.

[44]  T. Koepsell,et al.  Incidence of amyotrophic lateral sclerosis in three counties in western Washington state , 1996, Neurology.

[45]  C D Marsden,et al.  Alterations in striatal and extrastriatal D‐1 and D‐2 dopamine receptors in the MPTP‐treated common marmoset: An autoradiographic study , 1993, Synapse.

[46]  J. Haines,et al.  Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis , 1993, Nature.

[47]  W. O. Fenn,et al.  A barometric method for measuring ventilation in newborn infants. , 1955, Pediatrics.

[48]  A. Ludolph,et al.  Amyotrophic lateral sclerosis. , 2012, Current opinion in neurology.

[49]  A. Verma Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN , 2011 .

[50]  I. Martins,et al.  Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

[51]  R. Miller,et al.  Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). , 2003, Amyotrophic lateral sclerosis and other motor neuron disorders : official publication of the World Federation of Neurology, Research Group on Motor Neuron Diseases.

[52]  C. J. Maclean,et al.  Naturally occurring and experimentally induced beta-amyloid deposits in the brains of marmosets (Callithrix jacchus). , 2000, Journal of neural transmission.

[53]  T. Crow,et al.  Experimental induction of beta-amyloid plaques and cerebral angiopathy in primates. , 1993, Annals of the New York Academy of Sciences.