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The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

Nature reviews. Drug discovery, 2024-03, Vol.23 (3), p.157-158

2024

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Genethon: patient-empowered research

Expert opinion on orphan drugs, 2014-11, Vol.2 (11), p.1165-1170

Revah, Frédéric

2014

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Intrastriatal Injection of an Adenoviral Vector Expressing Glial-Cell-Line-Derived Neurotrophic Factor Prevents Dopaminergic Neuron Degeneration and Behavioral Impairment in a Rat Model of Parkinson Disease

Proceedings of the National Academy of Sciences - PNAS, 1997-08, Vol.94 (16), p.8818-8823

1997

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Design and synthesis of novel hydantoin-containing melanin-concentrating hormone receptor antagonists

Bioorganic & medicinal chemistry letters, 2007-07, Vol.17 (13), p.3754-3759

2007

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Prevention of motoneuron death by adenovirus-mediated neurotrophic factors

Journal of neuroscience research, 1997-05, Vol.48 (3), p.281-285

1997

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Adenovirus-mediated suicide gene transduction : Feasibility in lens epithelium and in prevention of posterior capsule opacification in rabbits

Human gene therapy, 1999-09, Vol.10 (14), p.2365-2372

1999

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The Noncompetitive Blocker [3H]Chlorpromazine Labels Three Amino Acids of the Acetylcholine Receptor γ Subunit: Implications for the α-Helical Organization of Regions MII and for the Structure of the Ion Channel

Proceedings of the National Academy of Sciences - PNAS, 1990-06, Vol.87 (12), p.4675-4679

1990

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Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases

Neuroreport, 1995-12, Vol.7 (1), p.373-378

1995

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G-Protein-Coupled Receptor Affinity Prediction Based on the Use of a Profiling Dataset: QSAR Design, Synthesis, and Experimental Validation

Journal of medicinal chemistry, 2005-10, Vol.48 (21), p.6563-6574

2005

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Allosteric Transitions of the Acetylcholine Receptor Probed at the Amino Acid Level with a Photolabile Cholinergic Ligand

Proceedings of the National Academy of Sciences - PNAS, 1991-06, Vol.88 (11), p.5051-5055

1991

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Neuromuscular function impairment is not caused by motor neurone loss in FALS mice: an electromyographic study

Neuroreport, 1996-05, Vol.7 (8), p.1427-1431

1996

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Refolding from denatured inclusion bodies, purification to homogeneity and simplified assay of MGDG synthases from land plants

Protein expression and purification, 2003-09, Vol.31 (1), p.79-87

2003

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Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases

Neuroreport, 1995-12, Vol.7 (1), p.373-373

1995

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Riluzole prolongs survival and delays muscle strength deterioration in mice with progressive motor neuronopathy ( pmn)

Journal of the neurological sciences, 2000-11, Vol.180 (1), p.55-61

2000

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Continuous delivery of neurotrophin 3 by gene therapy has a neuroprotective effect in experimental models of diabetic and acrylamide neuropathies

Human gene therapy, 2001-12, Vol.12 (18), p.2237-2249

2001

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Functional architecture of the nicotinic acetylcholine receptor: from electric organ to brain

Annual review of pharmacology and toxicology, 1991-01, Vol.31 (1), p.37-72

1991

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Partial prevention of cisplatin-induced neuropathy by electroporation-mediated nonviral gene transfer

Human gene therapy, 2001-03, Vol.12 (4), p.367-375

2001

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Identification of a novel amino acid alpha-tyrosine 93 within the cholinergic ligands-binding sites of the acetylcholine receptor by photoaffinity labeling. Additional evidence for a three-loop model of the cholinergic ligands-binding sites

The Journal of biological chemistry, 1990-06, Vol.265 (18), p.10430-10437

1990

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Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases

Neuroreport, 1995, Vol.6 (18), p.2473-2478

1995

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Functional significance of aromatic amino acids from three peptide loops of the α7 neuronal nicotinic receptor site investigated by site-directed mutagenesis

FEBS letters, 1991-12, Vol.294 (3), p.198-202

1991

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Calcium-Dependent Effect of the Thymic Polypeptide Thymopoietin on the Desensitization of the Nicotinic Acetylcholine Receptor

Proceedings of the National Academy of Sciences - PNAS, 1987-05, Vol.84 (10), p.3477-3481

1987

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QSAR Strategy and Experimental Validation for the Development of a GPCR Focused Library

QSAR & combinatorial science, 2005-06, Vol.24 (4), p.508-516

2005

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Immunohistochemical analysis of presenilin 2 expression in the mouse brain: distribution pattern and co-localization with presenilin 1 protein

Brain research, 1997-05, Vol.758 (1), p.209-217

1997

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Induction of Neuronal Apoptosis by Excitotoxins Associated with Long‐Lasting Increase of 12‐O‐Tetradecanoylphorbol 13‐Acetate‐Responsive Element‐Binding Activity

Journal of neurochemistry, 1995-09, Vol.65 (3), p.1027-1034

1995

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An adenovirus encoding CuZnSOD protects cultured striatal neurones against glutamate toxicity

Neuroreport, 1996-01, Vol.7 (2), p.497-501

1996

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The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

Genethon: patient-empowered research

Intrastriatal Injection of an Adenoviral Vector Expressing Glial-Cell-Line-Derived Neurotrophic Factor Prevents Dopaminergic Neuron Degeneration and Behavioral Impairment in a Rat Model of Parkinson Disease

Design and synthesis of novel hydantoin-containing melanin-concentrating hormone receptor antagonists

Prevention of motoneuron death by adenovirus-mediated neurotrophic factors

Adenovirus-mediated suicide gene transduction : Feasibility in lens epithelium and in prevention of posterior capsule opacification in rabbits

The Noncompetitive Blocker [3H]Chlorpromazine Labels Three Amino Acids of the Acetylcholine Receptor γ Subunit: Implications for the α-Helical Organization of Regions MII and for the Structure of the Ion Channel

Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases

G-Protein-Coupled Receptor Affinity Prediction Based on the Use of a Profiling Dataset: QSAR Design, Synthesis, and Experimental Validation

Allosteric Transitions of the Acetylcholine Receptor Probed at the Amino Acid Level with a Photolabile Cholinergic Ligand

Neuromuscular function impairment is not caused by motor neurone loss in FALS mice: an electromyographic study

Refolding from denatured inclusion bodies, purification to homogeneity and simplified assay of MGDG synthases from land plants

Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases

Riluzole prolongs survival and delays muscle strength deterioration in mice with progressive motor neuronopathy ( pmn)

Continuous delivery of neurotrophin 3 by gene therapy has a neuroprotective effect in experimental models of diabetic and acrylamide neuropathies

Functional architecture of the nicotinic acetylcholine receptor: from electric organ to brain

Partial prevention of cisplatin-induced neuropathy by electroporation-mediated nonviral gene transfer

Identification of a novel amino acid alpha-tyrosine 93 within the cholinergic ligands-binding sites of the acetylcholine receptor by photoaffinity labeling. Additional evidence for a three-loop model of the cholinergic ligands-binding sites

Specific and efficient gene transfer strategy offers new potentialities for the treatment of motor neurone diseases

Functional significance of aromatic amino acids from three peptide loops of the α7 neuronal nicotinic receptor site investigated by site-directed mutagenesis

Calcium-Dependent Effect of the Thymic Polypeptide Thymopoietin on the Desensitization of the Nicotinic Acetylcholine Receptor

QSAR Strategy and Experimental Validation for the Development of a GPCR Focused Library

Immunohistochemical analysis of presenilin 2 expression in the mouse brain: distribution pattern and co-localization with presenilin 1 protein

Induction of Neuronal Apoptosis by Excitotoxins Associated with Long‐Lasting Increase of 12‐O‐Tetradecanoylphorbol 13‐Acetate‐Responsive Element‐Binding Activity

An adenovirus encoding CuZnSOD protects cultured striatal neurones against glutamate toxicity

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