Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease.
about
Crystal Structure and Autoactivation Pathway of the Precursor Form of Human Tripeptidyl-peptidase 1, the Enzyme Deficient in Late Infantile Ceroid LipofuscinosisTherapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.Effective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brainAAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survivalDirected evolution of novel adeno-associated viruses for therapeutic gene delivery.Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).Dipeptidyl-peptidase I does not functionally compensate for the loss of tripeptidyl-peptidase I in the neurodegenerative disease late-infantile neuronal ceroid lipofuscinosis.Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis.Gene therapy for the nervous system: challenges and new strategies.Clinical applications involving CNS gene transfer.Gene therapy approaches for lysosomal storage disease: next-generation treatment.Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type AGemfibrozil and fenofibrate, Food and Drug Administration-approved lipid-lowering drugs, up-regulate tripeptidyl-peptidase 1 in brain cells via peroxisome proliferator-activated receptor α: implications for late infantile Batten disease therapy.Residual levels of tripeptidyl-peptidase I activity dramatically ameliorate disease in late-infantile neuronal ceroid lipofuscinosis.Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.Reversibility of neuropathology in Tay-Sachs-related diseases.Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease.The cellular pathology of lysosomal diseases.Viral vectors and delivery strategies for CNS gene therapy.Hydrogels for lentiviral gene delivery.Gene therapy for the neurological manifestations in lysosomal storage disorders.Multispecies-compatible antitumor effects of a cross-species small-interfering RNA against mammalian target of rapamycin.Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies.Timing of Gene Therapy Interventions: The Earlier, the Better.Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis.Widespread correction of central nervous system disease after intracranial gene therapy in a feline model of Sandhoff disease.Promising CNS-directed enzyme replacement therapy for lysosomal storage diseases.A zebrafish model of CLN2 disease is deficient in tripeptidyl peptidase 1 and displays progressive neurodegeneration accompanied by a reduction in proliferation.Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis.Inducible transgenic expression of tripeptidyl peptidase 1 in a mouse model of late-infantile neuronal ceroid lipofuscinosis.Gemfibrozil, food and drug administration-approved lipid-lowering drug, increases longevity in mouse model of late infantile neuronal ceroid lipofuscinosis.Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease.
P2860
Q27653009-954013B6-5D97-4598-B694-C1640CC250E5Q30541192-3B94CFEF-3776-40FE-85C1-7186DD8AECDFQ30573522-D30687D8-B781-4771-BFA0-4C089580736CQ33728295-0C846544-D854-478A-9BB4-33793D3951FEQ34188892-D23C0660-1324-4A0C-A547-24D025407D4EQ34349495-478037D6-450F-44D1-B355-D10212F3A414Q35158684-8FB980C4-9544-4B65-8D4E-8AD9B1FD011EQ35285680-1A5842BD-2F1C-4DF2-926C-A0CD4AFFA3BAQ35340196-FE2FB18D-1877-4508-8049-473EF7DDB963Q35899850-00D053C4-F286-465D-A182-155703CFDC92Q36147475-E3111AFD-6DF9-4D34-978F-6295A158DE2BQ36299764-A48E0BEF-B938-48BA-87F5-05D38296CE2DQ36385912-D4D86665-B853-4331-8C24-DACBCBEA11F9Q36772344-78C5D89D-DFDD-4086-9109-B24BE546F224Q36784566-A82D309C-1AAD-4665-871E-A976BE71E320Q37463989-DC588488-3C21-483E-95A5-59CD4375DB51Q37717896-E0736442-00BE-4722-8CDA-E1D37EB4E34CQ37944549-605CA493-A059-4AB7-B38F-57C63034A550Q38029645-CA038E46-4D06-40C1-A8C9-1E68C5B0D152Q38076509-84F429AB-7C4D-4D7F-A6C2-CAFA933047D1Q38200599-97D49975-0A13-4C92-B825-074BC71B0420Q39352883-A58E8AB9-E2EF-4D72-8455-9D3104DAC11FQ41785441-22EB288C-2CAA-44AE-B6EB-B789E72F34D1Q41939107-6A45E86C-E8C0-4D8D-A6FC-2508E11566FCQ41988291-25173945-795D-42AD-B651-06908D404383Q42177668-ECBCFBFE-9B52-4E06-AB64-08267B104B4EQ42768003-525529D2-5568-409F-B631-62B50DD4E4AEQ47073900-2A3CA0A4-8C12-409C-89F3-A5841FE788B0Q47548337-9FD93410-9B4E-4676-AB1B-95655479D02BQ48097209-3A37EBB0-8789-4EE5-BB76-5AED688B1D2FQ48234814-7B385969-D8D6-426B-A2CC-82118417A18EQ48633842-13224F87-8335-4517-AFAC-E09670E89374
P2860
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease.
description
2007 nî lūn-bûn
@nan
2007年の論文
@ja
2007年学术文章
@wuu
2007年学术文章
@zh
2007年学术文章
@zh-cn
2007年学术文章
@zh-hans
2007年学术文章
@zh-my
2007年学术文章
@zh-sg
2007年學術文章
@yue
2007年學術文章
@zh-hant
name
Timing of therapeutic interven ...... late infantile batten disease.
@en
Timing of therapeutic interven ...... late infantile batten disease.
@nl
type
label
Timing of therapeutic interven ...... late infantile batten disease.
@en
Timing of therapeutic interven ...... late infantile batten disease.
@nl
prefLabel
Timing of therapeutic interven ...... late infantile batten disease.
@en
Timing of therapeutic interven ...... late infantile batten disease.
@nl
P2093
P356
P1433
P1476
Timing of therapeutic interven ...... late infantile batten disease.
@en
P2093
Beverly L Davidson
Bradley L Hodges
David E Sleat
Eric M Roskelley
Istvan Sohar
James C Dodge
Lamya S Shihabuddin
Marco A Passini
Mario A Cabrera-Salazar
P304
P356
10.1038/SJ.MT.6300249
P577
2007-07-17T00:00:00Z