(Q45340805)

13 December 2017

Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis (English)

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Infantile neuronal ceroid lipofuscinosis

13 December 2017

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author

Jonathan D. Cooper

2

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13 December 2017

Michael Chang

1

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13 December 2017

David E Sleat

3

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13 December 2017

Seng H Cheng

4

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13 December 2017

James C Dodge

5

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13 December 2017

Marco A Passini

6

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13 December 2017

Peter Lobel

7

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13 December 2017

Beverly L Davidson

8

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13 December 2017

12 February 2008

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13 December 2017

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13 December 2017

16

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13 December 2017

4

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13 December 2017

649-656

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Current state of clinical and morphological features in human NCL.

13 December 2017

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7 January 2021

Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis

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7 January 2021

MRI of neuronal ceroid lipofuscinosis. II. Postmortem MRI and histopathological study of the brain in 16 cases of neuronal ceroid lipofuscinosis of juvenile or late infantile type.

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7 January 2021

A Case of Late Infantile Neuronal Ceroid Lipofuscinosis

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7 January 2021

Neuroradiological findings in classical late infantile neuronal ceroid-lipofuscinosis.

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7 January 2021

A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration

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7 January 2021

Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease

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7 January 2021

Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

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7 January 2021

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

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7 January 2021

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

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7 January 2021

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease

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7 January 2021

Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

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7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

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7 January 2021

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

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7 January 2021

Modification of the blood-brain barrier: increased concentration and fate of enzymes entering the brain

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7 January 2021

Delivery of hexosaminidase A to the cerebrum after osmotic modification of the blood--brain barrier

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7 January 2021

VEGF disrupts the neonatal blood-brain barrier and increases life span after non-ablative BMT in a murine model of congenital neurodegeneration caused by a lysosomal enzyme deficiency.

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7 January 2021

Convection perfusion of glucocerebrosidase for neuronopathic Gaucher's disease.

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7 January 2021

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I

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7 January 2021

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid

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7 January 2021

Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis

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7 January 2021

Glial activation spreads from specific cerebral foci and precedes neurodegeneration in presymptomatic ovine neuronal ceroid lipofuscinosis (CLN6).

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7 January 2021

Late onset neurodegeneration in the Cln3-/- mouse model of juvenile neuronal ceroid lipofuscinosis is preceded by low level glial activation.

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7 January 2021

Hippocampal pathology in the human neuronal ceroid-lipofuscinoses: distinct patterns of storage deposition, neurodegeneration and glial activation

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7 January 2021

Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta

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7 January 2021

Successful induction of immune tolerance to enzyme replacement therapy in canine mucopolysaccharidosis I

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7 January 2021

Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice

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7 January 2021

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

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7 January 2021

Identifiers

DOI

10.1038/MT.2008.9

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13 December 2017

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