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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Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral SclerosisIs spinal muscular atrophy a disease of the motor neurons only: pathogenesis and therapeutic implications?Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis.Gene delivery to the spinal cord using MRI-guided focused ultrasoundSomatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 SplicingAdeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model.Systemic injection of AAV9-GDNF provides modest functional improvements in the SOD1G93A ALS rat but has adverse side effects.Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.SMN deficiency disrupts gastrointestinal and enteric nervous system function in miceSMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA.AAV9 supports wide-scale transduction of the CNS and TDP-43 disease modeling in adult rats.A roadmap for precision medicine in the epilepsies.Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigsEfficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.Recombinant adeno-associated virus vectors in the treatment of rare diseases.Additive amelioration of ALS by co-targeting independent pathogenic mechanisms.Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.Optical stimulation for restoration of motor function after spinal cord injury.Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1.Spinal muscular atrophy--recent therapeutic advances for an old challenge.Developing therapies for spinal muscular atrophy.Subpial Adeno-associated Virus 9 (AAV9) Vector Delivery in Adult Mice.Viral vectors for therapy of neurologic diseases.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Advances in modeling and treating spinal muscular atrophyPost-modern therapeutic approaches for progressive myoclonus epilepsy.Translational development of splice-modifying antisense oligomers.Developmental regulation of SMN expression: pathophysiological implications and perspectives for therapy development in spinal muscular atrophy.Evaluation of intrathecal routes of administration for adeno-associated virus vectors in large animals.Improved gene delivery to adult mouse spinal cord through the use of engineered hybrid adeno-associated viral serotypes.Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.SMN Blood Levels in a Porcine Model of Spinal Muscular Atrophy.Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.Adeno Associated Virus 9-Based Gene Therapy Delivers a Functional Monocarboxylate Transporter 8, Improving Thyroid Hormone Availability to the Brain of Mct8-Deficient Mice.Gene therapy for GM1 gangliosidosis: challenges of translational medicineTransduction Profile of the Marmoset Central Nervous System Using Adeno-Associated Virus Serotype 9 Vectors.Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease.Survival Motor Neuron Protein is Released from Cells in Exosomes: A Potential Biomarker for Spinal Muscular Atrophy.Treatment Advances in Spinal Muscular Atrophy.
P2860
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P2860
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
description
2014 nî lūn-bûn
@nan
2014 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2014 թվականի հոտեմբերին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
name
Improving single injection CSF ...... in mice and nonhuman primates.
@ast
Improving single injection CSF ...... in mice and nonhuman primates.
@en
type
label
Improving single injection CSF ...... in mice and nonhuman primates.
@ast
Improving single injection CSF ...... in mice and nonhuman primates.
@en
prefLabel
Improving single injection CSF ...... in mice and nonhuman primates.
@ast
Improving single injection CSF ...... in mice and nonhuman primates.
@en
P2093
P2860
P50
P356
P1433
P1476
Improving single injection CSF ...... in mice and nonhuman primates.
@en
P2093
Brian K Kaspar
Jerry R Mendell
Julie Fitzgerald
Kathrin Meyer
Kevin D Foust
Leah Schmelzer
Lyndsey Braun
Olivia Michels
Pablo Morales
Shibi Likhite
P2860
P304
P356
10.1038/MT.2014.210
P577
2014-10-31T00:00:00Z