AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.
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Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression.Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders.Gene therapy for lysosomal storage disorders: a good start.Gene Therapy: The View from NCATS.Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease.Australian Cattle Dogs with Neuronal Ceroid Lipofuscinosis are Homozygous for a CLN5 Nonsense Mutation Previously Identified in Border Collies.Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors.Synergistic effects of treating the spinal cord and brain in CLN1 disease.Neuroinflammation as modifier of genetically caused neurological disorders of the central nervous system: Understanding pathogenesis and chances for treatment.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Perspective on innovative therapies for globoid cell leukodystrophy.Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal DegenerationA canine model for neuronal ceroid lipofuscinosis highlights the promise of gene therapy for lysosomal storage diseases.Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis.Canine neuronal ceroid lipofuscinoses: Promising models for preclinical testing of therapeutic interventions.AAVrh.10-mediated APOE2 CNS Gene Therapy for APOE4-associated Alzheimer's Disease.Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy.Fingolimod and Teriflunomide Attenuate Neurodegeneration in Mouse Models of Neuronal Ceroid Lipofuscinosis.Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease
P2860
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P2860
AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.
description
2015 nî lūn-bûn
@nan
2015 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած
@hyw
2015 թվականի նոյեմբերին հրատարակված գիտական հոդված
@hy
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
name
AAV gene transfer delays disea ...... fantile form of Batten disease
@nl
AAV gene transfer delays disea ...... antile form of Batten disease.
@ast
AAV gene transfer delays disea ...... antile form of Batten disease.
@en
type
label
AAV gene transfer delays disea ...... fantile form of Batten disease
@nl
AAV gene transfer delays disea ...... antile form of Batten disease.
@ast
AAV gene transfer delays disea ...... antile form of Batten disease.
@en
prefLabel
AAV gene transfer delays disea ...... fantile form of Batten disease
@nl
AAV gene transfer delays disea ...... antile form of Batten disease.
@ast
AAV gene transfer delays disea ...... antile form of Batten disease.
@en
P2093
P2860
P3181
P1476
AAV gene transfer delays disea ...... antile form of Batten disease.
@en
P2093
Beverly L Davidson
Elena Lysenko
Fred A Wininger
Gayle C Johnson
Joan R Coates
Luis Tecedor
Martin L Katz
Rebecca E H Whiting
Whitney M Young
Yonghong Chen
P2860
P304
P3181
P356
10.1126/SCITRANSLMED.AAC6191
P407
P577
2015-11-01T00:00:00Z