Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease

scientific article published on 31 March 2016

Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1016/J.EXER.2016.03.023
P932PMC publication ID4957944
P698PubMed publication ID27039708

P2093author name stringMartin L Katz
Rebecca E H Whiting
Cheryl A Jensen
Jacqueline W Pearce
Lauren E Gillespie
Daniel E Bristow
P2860cites workAAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten diseaseQ27324755
Characterization of thoracic motor and sensory neurons and spinal nerve roots in canine degenerative myelopathy, a potential disease model of amyotrophic lateral sclerosisQ33894360
Characterization of intercostal muscle pathology in canine degenerative myelopathy: a disease model for amyotrophic lateral sclerosisQ33894394
Genetic basis and phenotypic correlations of the neuronal ceroid lipofusinoses.Q34336077
Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).Q34679086
Multifocal retinopathy in Dachshunds with CLN2 neuronal ceroid lipofuscinosisQ35588896
Genetics of the neuronal ceroid lipofuscinoses (Batten disease).Q36053005
Quantitative assessment of the canine pupillary light reflexQ37095330
Pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosisQ37353812
Neuronal ceroid lipofuscinosis: impact of recent genetic advances and expansion of the clinicopathologic spectrumQ38115191
Lysosomal storage diseases: from pathophysiology to therapy.Q38318000
Golden Retriever dogs with neuronal ceroid lipofuscinosis have a two-base-pair deletion and frameshift in CLN5.Q38386636
Routes for Drug Delivery to the Eye and Retina: Intravitreal Injections.Q38616394
A frame shift mutation in canine TPP1 (the ortholog of human CLN2) in a juvenile Dachshund with neuronal ceroid lipofuscinosisQ40333380
Assessment of retinal function and characterization of lysosomal storage body accumulation in the retinas and brains of Tibetan Terriers with ceroid-lipofuscinosisQ45250738
Retinal pathology in a canine model of late infantile neuronal ceroid lipofuscinosisQ46698740
Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosisQ48513500
Enzyme replacement therapy delays pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosisQ48702687
Selective presence of acid hydrolases in the interphotoreceptor matrixQ69426514
Proteins of the bovine interphotoreceptor matrix: retinoid binding and other functionsQ69870716
Lysosomal enzymes in the interphotoreceptor matrix: acid proteaseQ71483149
Guidelines for clinical electroretinography in the dog: 2012 updateQ86885801
P921main subjectgene therapyQ213901
retinal ganglion cellQ927337
P304page(s)276-282
P577publication date2016-03-31
P1433published inExperimental Eye ResearchQ15754753
P1476titleIntracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease
P478volume146

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cites work (P2860)
Q42370349A canine model for neuronal ceroid lipofuscinosis highlights the promise of gene therapy for lysosomal storage diseases
Q92122633A mixed breed dog with neuronal ceroid lipofuscinosis is homozygous for a CLN5 nonsense mutation previously identified in Border Collies and Australian Cattle Dogs
Q33782257CLINICAL PROGRESS IN INHERITED RETINAL DEGENERATIONS: GENE THERAPY CLINICAL TRIALS AND ADVANCES IN GENETIC SEQUENCING.
Q47786937Canine neuronal ceroid lipofuscinoses: Promising models for preclinical testing of therapeutic interventions
Q90175159Emerging treatments for progressive myoclonus epilepsies
Q30846558Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression
Q90441214Mice deficient in the lysosomal enzyme palmitoyl-protein thioesterase 1 (PPT1) display a complex retinal phenotype

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