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Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
- Published in:
- Nature Biotechnology, 2022, v. 40, n. 11, p. 1692, doi. 10.1038/s41587-022-01497-7
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- Article
Astrocytes from familial and sporadic ALS patients are toxic to motor neurons.
- Published in:
- Nature Biotechnology, 2011, v. 29, n. 9, p. 824, doi. 10.1038/nbt.1957
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- Article
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
- Published in:
- Nature Biotechnology, 2010, v. 28, n. 3, p. 271, doi. 10.1038/nbt.1610
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- Article
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.
- Published in:
- Nature Biotechnology, 2009, v. 27, n. 1, p. 59, doi. 10.1038/nbt.1515
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- Article
A role for glia in the progression of Rett's syndrome.
- Published in:
- Nature, 2011, v. 475, n. 7357, p. 497, doi. 10.1038/nature10214
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- Article
Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose-response Study in Mice and Nonhuman Primates.
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- Molecular Therapy, 2015, v. 23, n. 3, p. 477, doi. 10.1038/mt.2014.210
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- Article
Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS.
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- Molecular Therapy, 2013, v. 21, n. 12, p. 2148, doi. 10.1038/mt.2013.211
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- Article
Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA.
- Published in:
- Human Molecular Genetics, 2015, v. 24, n. 21, p. 6160, doi. 10.1093/hmg/ddv332
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- Article
SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA.
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- Human Molecular Genetics, 2015, v. 24, n. 19, p. 5524, doi. 10.1093/hmg/ddv283
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- Article
SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice.
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- Human Molecular Genetics, 2015, v. 24, n. 13, p. 3847, doi. 10.1093/hmg/ddv127
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- Article
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.
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- Human Molecular Genetics, 2012, v. 21, n. 7, p. 1625, doi. 10.1093/hmg/ddr600
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- Article
Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 11, p. 1971, doi. 10.1038/mt.2011.157
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- Publication type:
- Article
Nigrostriatal rAAV-mediated GDNF Overexpression Induces Robust Weight Loss in a Rat Model of Age-related Obesity.
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- Molecular Therapy, 2009, v. 17, n. 6, p. 980, doi. 10.1038/mt.2009.45
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- Article
Recombinant Adeno-associated Viral Vectors as Therapeutic Agents to Treat Neurological Disorders.
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- Molecular Therapy, 2006, v. 13, n. 3, p. 463, doi. 10.1016/j.ymthe.2005.11.009
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- Article
654. Quantifying GDNF Levels in the Spinal Cord after Intracerebral Delivery of rAAV5 GDNF
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- 2005
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- Abstract
Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.
- Published in:
- Human Molecular Genetics, 2010, v. 19, n. 20, p. 3895, doi. 10.1093/hmg/ddq300
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- Publication type:
- Article
Intravenous AAV9 efficiently transduces myenteric neurons in neonate and juvenile mice.
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- Frontiers in Molecular Neuroscience, 2014, v. 7, p. 1, doi. 10.3389/fnmol.2014.00081
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- Article
Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3).
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- Journal of Neuroscience, 2016, v. 36, n. 37, p. 9669, doi. 10.1523/JNEUROSCI.1635-16.2016
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- Article
Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome.
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- Journal of Neuroscience, 2013, v. 33, n. 34, p. 13612, doi. 10.1523/JNEUROSCI.1854-13.2013
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- Publication type:
- Article