Therapeutic rAAVrh 10 Mediated SOD 1 Silencing in Adult SOD 1 ( G 93 A ) Mice and Nonhuman Primates
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Robert H. Brown | C. Mueller | M. ElMallah | G. Gao | Lina Song | Q. Su | F. Borel | Brynn H. Cardozo | Jake P. Metterville | G. Gernoux | G. Cabrera | Robert H. Brown | C. Mueller | Gwladys Gernoux
[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.