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zadetkov: 181
1.
  • Adeno-associated virus-deli... Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model
    Stoica, Lorelei; Todeasa, Sophia H.; Cabrera, Gabriela Toro ... Annals of neurology, April 2016, Letnik: 79, Številka: 4
    Journal Article
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    Objective Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by loss of motor neurons, resulting in progressive muscle weakness, paralysis, and death within 5 ...
Celotno besedilo
Dostopno za: UL

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2.
  • Mutation in protein disulfi... Mutation in protein disulfide isomerase A3 causes neurodevelopmental defects by disturbing endoplasmic reticulum proteostasis
    Bilches Medinas, Danilo; Malik, Sajid; Yıldız‐Bölükbaşı, Esra ... EMBO journal, 12/2022, Letnik: 41, Številka: 2
    Journal Article
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    Recessive gene mutations underlie many developmental disorders and often lead to disabling neurological problems. Here, we report identification of a homozygous c.170G>A (p.Cys57Tyr or C57Y) mutation ...
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Dostopno za: UL
3.
  • Spinal projections of the A... Spinal projections of the A5, A6 (locus coeruleus), and A7 noradrenergic cell groups in rats
    Bruinstroop, Eveline; Cano, Georgina; Vanderhorst, Veronique G.J.M. ... Journal of comparative neurology, 15 June 2012, Letnik: 520, Številka: 9
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    The pontine noradrenergic cell groups, A5, A6 (locus coeruleus), and A7, provide the only noradrenergic innervation of the spinal cord, but the individual contribution of each of these populations to ...
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Dostopno za: UL

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4.
  • Intracranial AAV‐IFN‐β gene... Intracranial AAV‐IFN‐β gene therapy eliminates invasive xenograft glioblastoma and improves survival in orthotopic syngeneic murine model
    GuhaSarkar, Dwijit; Neiswender, James; Su, Qin ... Molecular oncology, February 2017, Letnik: 11, Številka: 2
    Journal Article
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    The highly invasive property of glioblastoma (GBM) cells and genetic heterogeneity are largely responsible for tumor recurrence after the current standard‐of‐care treatment and thus a direct cause of ...
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Dostopno za: UL

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5.
  • Viral vectors for therapy o... Viral vectors for therapy of neurologic diseases
    Choudhury, Sourav R.; Hudry, Eloise; Maguire, Casey A. ... Neuropharmacology, 07/2017, Letnik: 120
    Journal Article
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    Neurological disorders – disorders of the brain, spine and associated nerves – are a leading contributor to global disease burden with a shockingly large associated economic cost. Various treatment ...
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Dostopno za: UL

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6.
  • Life‐Limiting Peripheral Or... Life‐Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy
    Johnson, Aime K.; McCurdy, Victoria J.; Gray‐Edwards, Heather L. ... Annals of neurology, November 2023, 2023-11-00, 20231101, Letnik: 94, Številka: 5
    Journal Article
    Recenzirano

    Objective GM2 gangliosidosis is usually fatal by 5 years of age in its 2 major subtypes, Tay‐Sachs and Sandhoff disease. First reported in 1881, GM2 gangliosidosis has no effective treatment today, ...
Celotno besedilo
Dostopno za: UL
7.
  • A divalent siRNA chemical s... A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
    Alterman, Julia F; Godinho, Bruno M D C; Hassler, Matthew R ... Nature biotechnology, 08/2019, Letnik: 37, Številka: 8
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    Sustained silencing of gene expression throughout the brain using small interfering RNAs (siRNAs) has not been achieved. Here we describe an siRNA architecture, divalent siRNA (di-siRNA), that ...
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Dostopno za: UL

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8.
  • Cerebral organoids derived ... Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]
    Allende, Maria L.; Cook, Emily K.; Larman, Bridget C. ... Journal of lipid research, 03/2018, Letnik: 59, Številka: 3
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    Sandhoff disease, one of the GM2 gangliosidoses, is a lysosomal storage disorder characterized by the absence of β-hexosaminidase A and B activity and the concomitant lysosomal accumulation of its ...
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Dostopno za: UL

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9.
  • Several rAAV Vectors Effici... Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System
    Zhang, Hongwei; Yang, Bin; Mu, Xin ... Molecular therapy, 08/2011, Letnik: 19, Številka: 8
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    Noninvasive systemic gene delivery to the central nervous system (CNS) has largely been impeded by the blood–brain barrier (BBB). Recent studies documented widespread CNS gene transfer after ...
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Dostopno za: UL

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10.
  • Microvesicle-associated AAV... Microvesicle-associated AAV Vector as a Novel Gene Delivery System
    Maguire, Casey A; Balaj, Leonora; Sivaraman, Sarada ... Molecular therapy, 05/2012, Letnik: 20, Številka: 5
    Journal Article
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    Adeno-associated virus (AAV) vectors have shown remarkable efficiency for gene delivery to cultured cells and in animal models of human disease. However, limitations to AAV vectored gene transfer ...
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Dostopno za: UL

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zadetkov: 181

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