A direct gene transfer strategy via brain internal capsule reverses the biochemical defect in Tay-Sachs disease
about
Development of a New Tool for 3D Modeling for Regenerative MedicineGene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flowIn cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 gangliosideMethods for gene transfer to the central nervous system.Pharmacotherapeutic strategies using small molecules for the treatment of glycolipid lysosomal storage disorders.MicroRNA implications across neurodevelopment and neuropathology.Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.HSV Recombinant Vectors for Gene Therapy.Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.Mechanotransduction: tuning stem cells fate.Stem Cells in Neurological Disorders: Emerging Therapy with Stunning Hopes.Perspective on innovative therapies for globoid cell leukodystrophy.Intraneural GJB1 gene delivery improves nerve pathology in a model of X-linked Charcot-Marie-Tooth disease.Gene delivery targeted to oligodendrocytes using a lentiviral vector.Β-hexosaminidase over-expression affects lysosomal glycohydrolases expression and glycosphingolipid metabolism in mammalian cells.Delivering drugs to the central nervous system: an overview.Evaluation and optimization of the administration of a selectively replicating herpes simplex viral vector to the brain by convection-enhanced delivery.Patented therapeutic RNAi strategies for neurodegenerative diseases of the CNS
P2860
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P2860
A direct gene transfer strategy via brain internal capsule reverses the biochemical defect in Tay-Sachs disease
description
2005 nî lūn-bûn
@nan
2005 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2005 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2005年の論文
@ja
2005年論文
@yue
2005年論文
@zh-hant
2005年論文
@zh-hk
2005年論文
@zh-mo
2005年論文
@zh-tw
2005年论文
@wuu
name
A direct gene transfer strateg ...... al defect in Tay-Sachs disease
@ast
A direct gene transfer strateg ...... al defect in Tay-Sachs disease
@en
type
label
A direct gene transfer strateg ...... al defect in Tay-Sachs disease
@ast
A direct gene transfer strateg ...... al defect in Tay-Sachs disease
@en
prefLabel
A direct gene transfer strateg ...... al defect in Tay-Sachs disease
@ast
A direct gene transfer strateg ...... al defect in Tay-Sachs disease
@en
P2093
P50
P356
P1476
A direct gene transfer strateg ...... al defect in Tay-Sachs disease
@en
P2093
A Orlacchio
C Bordignon
K Sandhoff
M G Cusella De Angelis
P304
P356
10.1093/HMG/DDI216
P50
P577
2005-06-16T00:00:00Z