Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis.

Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis.

Item
http://www.wikidata.org/entity/Q33939283
Moving towards effective therapeutic strategies for Neuronal Ceroid LipofuscinosisTreatment of the Ppt1(-/-) mouse model of infantile neuronal ceroid lipofuscinosis with the N-methyl-D-aspartate (NMDA) receptor antagonist memantine.An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosisPathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).Reversible Cysteine Acylation Regulates the Activity of Human Palmitoyl-Protein Thioesterase 1 (PPT1).Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis.Histochemical localization of palmitoyl protein thioesterase-1 activityConsiderations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease)Synergistic effects of treating the spinal cord and brain in CLN1 disease.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Widespread Expression of a Membrane-Tethered Version of the Soluble Lysosomal Enzyme Palmitoyl Protein Thioesterase-1.
P2860

Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis.

Item
http://www.wikidata.org/entity/Q33939283
description
2012 nî lūn-bûn
@nan
2012 թուականի Փետրուարին հրատարակուած գիտական յօդուած
@hyw
2012 թվականի փետրվարին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
Combination small molecule PPT ...... euronal ceroid lipofuscinosis.
@ast
Combination small molecule PPT ...... euronal ceroid lipofuscinosis.
@en
type
label
Combination small molecule PPT ...... euronal ceroid lipofuscinosis.
@ast
Combination small molecule PPT ...... euronal ceroid lipofuscinosis.
@en
prefLabel
Combination small molecule PPT ...... euronal ceroid lipofuscinosis.
@ast
Combination small molecule PPT ...... euronal ceroid lipofuscinosis.
@en
P1433
P1476
P2093
P2860
P2888
P304
P31
P356
P433
P478
P577
P6179
P698
P921
P932
P356
P1433
P1476
Combination small molecule PPT ...... euronal ceroid lipofuscinosis.
@en
P2093
P2860
P2888
P304
P31
P356
P433
P478
P577
P6179
P698
P921
P932