Search Results - "Deglon, N"

Gene editing as a therapeutic strategy for spinocerebellar ataxia type-3 by Déglon, N.

“…Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, is a neurodegenerative disease caused by expanded polyglutamine repeats in exon 10…”
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Calpastatin-mediated inhibition of calpains in the mouse brain prevents mutant ataxin 3 proteolysis, nuclear localization and aggregation, relieving Machado―Joseph disease by SIMOES, Ana T, GONCALVES, Nélio, KOEPPEN, Arnulf, DEGLON, Nicole, KÜGLER, Sebastian, DUARTE, Carlos Bandeira, DE ALMEIDA, Luís Pereira

“…Machado-Joseph disease is the most frequently found dominantly-inherited cerebellar ataxia. Over-repetition of a CAG trinucleotide in the MJD1 gene translates…”
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Viral vectors as tools to model and treat neurodegenerative disorders by Déglon, N., Hantraye, P.

“…The identification of disease‐causing genes in familial forms of neurodegenerative disorders and the development of genetic models closely replicating human…”
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Lentiviruses as vectors for CNS diseases by Déglon, N, Aebischer, P

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AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease by Miniarikova, J, Zimmer, V, Martier, R, Brouwers, C C, Pythoud, C, Richetin, K, Rey, M, Lubelski, J, Evers, M M, van Deventer, S J, Petry, H, Déglon, N, Konstantinova, P

“…Huntington’s disease (HD) is a fatal progressive neurodegenerative disorder caused by a mutation in the huntingtin ( HTT ) gene. To date, there is no treatment…”
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BDNF overexpression in mouse hippocampal astrocytes promotes local neurogenesis and elicits anxiolytic-like activities by Quesseveur, G, David, D J, Gaillard, M C, Pla, P, Wu, M V, Nguyen, H T, Nicolas, V, Auregan, G, David, I, Dranovsky, A, Hantraye, P, Hen, R, Gardier, A M, Déglon, N, Guiard, B P

“…The therapeutic activity of selective serotonin (5-HT) reuptake inhibitors (SSRIs) relies on long-term adaptation at pre- and post-synaptic levels. The…”
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Gene transfer engineering for astrocyte-specific silencing in the CNS by Merienne, N, Delzor, A, Viret, A, Dufour, N, Rey, M, Hantraye, P, Déglon, N

“…Cell-type-specific gene silencing is critical to understand cell functions in normal and pathological conditions, in particular in the brain where strong…”
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Formation of hippocampal mHTT aggregates leads to impaired spatial memory, hippocampal activation and adult neurogenesis by Schwab, LC, Richetin, K, Barker, RA, Deglon, N

“…Abstract Huntington's disease (HD) is a genetic neurodegenerative disorder characterized by a triad of motor, psychiatric and cognitive deficits with the…”
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Optimization of human erythropoietin secretion from MLV-infected human primary fibroblasts used for encapsulated cell therapy by Schwenter, F., Déglon, N., Aebischer, P.

“…Background The transplantation of encapsulated cells genetically engineered to secrete human erythropoietin (hEpo) represents an alternative to repeated…”
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Activity analysis of housekeeping promoters using self-inactivating lentiviral vector delivery into the mouse retina by KOSTIC, C, CHIODINI, F, ARSENIJEVIC, Y, SALMON, P, WIZNEROWICZ, M, DEGLON, N, HORNFELD, D, TRONO, D, AEBISCHER, P, SCHORDERET, D. F, MUNIER, F. L

“…For most retinal degeneration disorders, no efficient treatment exists to preserve photoreceptors (PRs) and, consequently, to maintain vision. Gene transfer…”
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Implication of the JNK pathway in a rat model of Huntington's disease by Perrin, V., Dufour, N., Raoul, C., Hassig, R., Brouillet, E., Aebischer, P., Luthi-Carter, R., Déglon, N.

“…Huntington's disease (HD) is a neurodegenerative disorder resulting from the expansion of a glutamine repeat (polyQ) in the N-terminus of the huntingtin (htt)…”
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α-Synucleinopathy and Selective Dopaminergic Neuron Loss in a Rat Lentiviral-Based Model of Parkinson's Disease by Lo Bianco, C., J-L. Ridet, Schneider, B. L., Déglon, N., Aebischer, P.

“…Parkinson's disease (PD) is characterized by the progressive loss of substantia nigra dopaminergic neurons and the presence of cytoplasmic inclusions named…”
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Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study by Bloch, J, Bachoud-Lévi, A C, Déglon, N, Lefaucheur, J P, Winkel, L, Palfi, S, Nguyen, J P, Bourdet, C, Gaura, V, Remy, P, Brugières, P, Boisse, M-F, Baudic, S, Cesaro, P, Hantraye, P, Aebischer, P, Peschanski, M

“…Huntington's disease (HD) is a monogenic neurodegenerative disease that affects the efferent neurons of the striatum. The protracted evolution of the pathology…”
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Lentiviral nigral delivery of GDNF does not prevent neurodegeneration in a genetic rat model of Parkinson's disease by Lo Bianco, C., Déglon, N., Pralong, W., Aebischer, P.

“…Viral delivery of glial cell line-derived neurotrophic factor (GDNF) currently represents one of the most promising neuroprotective strategies for Parkinson's…”
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Efficient Gene Transfer and Expression of Biologically Active Glial Cell Line‐Derived Neurotrophic Factor in Rat Motoneurons Transduced with Lentiviral Vectors by Cisterni, C., Henderson, C. E., Aebischer, P., Pettmann, B., Déglon, N.

“…: Several studies have shown the ability of human immunodeficiency virus type 1 (HIV1)‐based lentiviral vectors to infect nondividing brain and retinal neurons…”
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Neuroprotective Effect of a CNTF-Expressing Lentiviral Vector in the Quinolinic Acid Rat Model of Huntington's Disease by de Almeida, L.Pereira, Zala, D., Aebischer, P., Déglon, N.

“…Neurodegenerative diseases represent promising targets for gene therapy approaches provided effective transfer vectors. In the present study, we evaluated the…”
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Improvement of mouse β-thalassemia upon erythropoietin delivery by encapsulated myoblasts by DALLE, B, PAYEN, E, REGULIER, E, DEGLON, N, ROUYER-FESSARD, P, BEUZARD, Y, AEBISCHER, P

“…The goal of the present study was to analyze if sustained delivery of elevated doses of recombinant erythropoietin (Epo), by genetically modified and…”
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Gene therapy for amyotrophic lateral sclerosis (ALS) using a polymer encapsulated xenogenic cell line engineered to secrete hCNTF by Aebischer, P, Pochon, N A, Heyd, B, Deglon, N, Joseph, J M, Zurn, A D, Baetge, E E, Hammang, J P, Goddard, M, Lysaght, M, Kaplan, F, Kato, A C, Schluep, M, Hirt, L, Regli, F, Porchet, F, De Tribolet, N

“…The gene therapy approach presented in this protocol employs a polymer encapsulated, xenogenic, transfected cell line to release human ciliary neurotrophic…”
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Dose-dependent neuroprotective effect of ciliary neurotrophic factor delivered via tetracycline-regulated lentiviral vectors in the quinolinic acid rat model of Huntington's disease by Régulier, E, Pereira de Almeida, L, Sommer, B, Aebischer, P, Déglon, N

“…The ability to regulate gene expression constitutes a prerequisite for the development of gene therapy strategies aimed at the treatment of neurologic…”
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The JAK/STAT3 pathway is a common inducer of astrocyte reactivity in Alzheimer's and Huntington's diseases by Ben Haim, Lucile, Ceyzériat, Kelly, Carrillo-de Sauvage, Maria Angeles, Aubry, Fabien, Auregan, Gwennaëlle, Guillermier, Martine, Ruiz, Marta, Petit, Fanny, Houitte, Diane, Faivre, Emilie, Vandesquille, Matthias, Aron-Badin, Romina, Dhenain, Marc, Déglon, Nicole, Hantraye, Philippe, Brouillet, Emmanuel, Bonvento, Gilles, Escartin, Carole

“…Astrocyte reactivity is a hallmark of neurodegenerative diseases (ND), but its effects on disease outcomes remain highly debated. Elucidation of the signaling…”
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