{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,25]],"date-time":"2026-03-25T07:05:44Z","timestamp":1774422344301,"version":"3.50.1"},"reference-count":74,"publisher":"Public Library of Science (PLoS)","issue":"1","license":[{"start":{"date-parts":[[2016,1,5]],"date-time":"2016-01-05T00:00:00Z","timestamp":1451952000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"DOI":"10.1371\/journal.pone.0146466","type":"journal-article","created":{"date-parts":[[2016,1,5]],"date-time":"2016-01-05T19:19:14Z","timestamp":1452021554000},"page":"e0146466","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":34,"title":["Reversible Cysteine Acylation Regulates the Activity of Human Palmitoyl-Protein Thioesterase 1 (PPT1)"],"prefix":"10.1371","volume":"11","author":[{"given":"Michal","family":"Segal-Salto","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tamar","family":"Sapir","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Orly","family":"Reiner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"340","published-online":{"date-parts":[[2016,1,5]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1002\/ajmg.1320310606","article-title":"Batten disease: past, present, and future","volume":"5","author":"JA Rider","year":"1988","journal-title":"Am J Med Genet Suppl"},{"issue":"24","key":"ref2","doi-asserted-by":"crossref","first-page":"13566","DOI":"10.1073\/pnas.251485198","article-title":"Disruption of PPT1 or PPT2 causes neuronal ceroid lipofuscinosis in knockout mice","volume":"98","author":"P Gupta","year":"2001","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"4","key":"ref3","doi-asserted-by":"crossref","first-page":"697","DOI":"10.1016\/j.bbamcr.2008.11.004","article-title":"Neuronal ceroid lipofuscinoses","volume":"1793","author":"A Jalanko","year":"2009","journal-title":"Biochim Biophys Acta"},{"issue":"7","key":"ref4","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1016\/j.braindev.2008.12.008","article-title":"Towards understanding the neuronal ceroid lipofuscinoses","volume":"31","author":"A Kohlschutter","year":"2009","journal-title":"Brain Dev"},{"issue":"6541","key":"ref5","doi-asserted-by":"crossref","first-page":"584","DOI":"10.1038\/376584a0","article-title":"Mutations in the palmitoyl protein thioesterase gene causing infantile neuronal ceroid lipofuscinosis","volume":"376","author":"J Vesa","year":"1995","journal-title":"Nature"},{"issue":"2","key":"ref6","first-page":"291","article-title":"Mutations in the palmitoyl-protein thioesterase gene (PPT; CLN1) causing juvenile neuronal ceroid lipofuscinosis with granular osmiophilic deposits","volume":"7","author":"HM Mitchison","year":"1998","journal-title":"Hum Mol Genet"},{"issue":"9","key":"ref7","doi-asserted-by":"crossref","first-page":"4573","DOI":"10.1073\/pnas.080508097","article-title":"The crystal structure of palmitoyl protein thioesterase 1 and the molecular basis of infantile neuronal ceroid lipofuscinosis","volume":"97","author":"JJ Bellizzi 3rd","year":"2000","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"2","key":"ref8","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1016\/j.molcel.2011.01.001","article-title":"FKBP12 binds to acylated h-ras and promotes depalmitoylation","volume":"41","author":"IM Ahearn","year":"2011","journal-title":"Mol Cell"},{"issue":"1","key":"ref9","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1038\/emboj.2009.325","article-title":"Ndel1 palmitoylation: a new mean to regulate cytoplasmic dynein activity","volume":"29","author":"A Shmueli","year":"2010","journal-title":"EMBO J"},{"issue":"10","key":"ref10","doi-asserted-by":"crossref","first-page":"791","DOI":"10.1038\/nrn940","article-title":"Protein palmitoylation: a regulator of neuronal development and function","volume":"3","author":"Ael D El-Husseini","year":"2002","journal-title":"Nat Rev Neurosci"},{"issue":"5","key":"ref11","doi-asserted-by":"crossref","first-page":"1003","DOI":"10.1016\/j.cell.2006.03.042","article-title":"Global analysis of protein palmitoylation in yeast","volume":"125","author":"AF Roth","year":"2006","journal-title":"Cell"},{"issue":"2","key":"ref12","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1038\/nmeth.1293","article-title":"Large-scale profiling of protein palmitoylation in mammalian cells","volume":"6","author":"BR Martin","year":"2009","journal-title":"Nat Methods"},{"issue":"7224","key":"ref13","doi-asserted-by":"crossref","first-page":"904","DOI":"10.1038\/nature07605","article-title":"Neural palmitoyl-proteomics reveals dynamic synaptic palmitoylation","volume":"456","author":"R Kang","year":"2008","journal-title":"Nature"},{"issue":"1\u20132","key":"ref14","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1016\/S0005-2760(98)00130-1","article-title":"Signalling functions of protein palmitoylation","volume":"1436","author":"JT Dunphy","year":"1998","journal-title":"Biochim Biophys Acta"},{"issue":"1","key":"ref15","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0167-4889(99)00075-0","article-title":"Fatty acylation of proteins: new insights into membrane targeting of myristoylated and palmitoylated proteins","volume":"1451","author":"MD Resh","year":"1999","journal-title":"Biochim Biophys Acta"},{"issue":"6","key":"ref16","first-page":"901","article-title":"Greasing transmission: palmitoylation at the synapse","volume":"44","author":"P Washbourne","year":"2004","journal-title":"Neuron"},{"issue":"1","key":"ref17","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1038\/nrm2084","article-title":"Palmitoylation: policing protein stability and traffic","volume":"8","author":"ME Linder","year":"2007","journal-title":"Nat Rev Mol Cell Biol"},{"issue":"3","key":"ref18","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1038\/nrn2788","article-title":"Protein palmitoylation in neuronal development and synaptic plasticity","volume":"11","author":"Y Fukata","year":"2010","journal-title":"Nat Rev Neurosci"},{"issue":"6","key":"ref19","doi-asserted-by":"crossref","first-page":"987","DOI":"10.1016\/j.neuron.2004.12.005","article-title":"Identification of PSD-95 palmitoylating enzymes","volume":"44","author":"M Fukata","year":"2004","journal-title":"Neuron"},{"issue":"19","key":"ref20","doi-asserted-by":"crossref","first-page":"5240","DOI":"10.1002\/j.1460-2075.1996.tb00909.x","article-title":"Human palmitoyl protein thioesterase: evidence for lysosomal targeting of the enzyme and disturbed cellular routing in infantile neuronal ceroid lipofuscinosis","volume":"15","author":"E Hellsten","year":"1996","journal-title":"EMBO J"},{"issue":"26","key":"ref21","doi-asserted-by":"crossref","first-page":"15831","DOI":"10.1074\/jbc.271.26.15831","article-title":"Lysosomal targeting of palmitoyl-protein thioesterase","volume":"271","author":"LA Verkruyse","year":"1996","journal-title":"J Biol Chem"},{"issue":"7","key":"ref22","doi-asserted-by":"crossref","first-page":"1352","DOI":"10.1194\/jlr.R600010-JLR200","article-title":"Thematic review series: lipid posttranslational modifications. Lysosomal metabolism of lipid-modified proteins","volume":"47","author":"JY Lu","year":"2006","journal-title":"J Lipid Res"},{"issue":"4","key":"ref23","doi-asserted-by":"crossref","first-page":"686","DOI":"10.1074\/mcp.M500343-MCP200","article-title":"Identification of sites of mannose 6-phosphorylation on lysosomal proteins","volume":"5","author":"DE Sleat","year":"2006","journal-title":"Mol Cell Proteomics"},{"issue":"32","key":"ref24","doi-asserted-by":"crossref","first-page":"19191","DOI":"10.1074\/jbc.271.32.19191","article-title":"Rat brain contains high levels of mannose-6-phosphorylated glycoproteins including lysosomal enzymes and palmitoyl-protein thioesterase, an enzyme implicated in infantile neuronal lipofuscinosis","volume":"271","author":"DE Sleat","year":"1996","journal-title":"J Biol Chem"},{"issue":"3","key":"ref25","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1038\/nrm1050","article-title":"Mannose 6-phosphate receptors: new twists in the tale","volume":"4","author":"P Ghosh","year":"2003","journal-title":"Nat Rev Mol Cell Biol"},{"issue":"9","key":"ref26","doi-asserted-by":"crossref","first-page":"1540","DOI":"10.1016\/j.yexcr.2006.01.034","article-title":"Palmitoyl protein thioesterase 1 (PPT1) deficiency causes endocytic defects connected to abnormal saposin processing","volume":"312","author":"L Ahtiainen","year":"2006","journal-title":"Exp Cell Res"},{"issue":"11","key":"ref27","doi-asserted-by":"crossref","first-page":"1826","DOI":"10.1093\/hmg\/ddl105","article-title":"Endoplasmic reticulum stress-induced caspase-4 activation mediates apoptosis and neurodegeneration in INCL","volume":"15","author":"SJ Kim","year":"2006","journal-title":"Hum Mol Genet"},{"issue":"2","key":"ref28","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1093\/hmg\/ddi451","article-title":"Palmitoyl-protein thioesterase-1 deficiency mediates the activation of the unfolded protein response and neuronal apoptosis in INCL","volume":"15","author":"Z Zhang","year":"2006","journal-title":"Hum Mol Genet"},{"issue":"3","key":"ref29","doi-asserted-by":"crossref","first-page":"368","DOI":"10.1002\/cne.10492","article-title":"Palmitoyl protein thioesterase 1 is targeted to the axons in neurons","volume":"455","author":"L Ahtiainen","year":"2003","journal-title":"J Comp Neurol"},{"issue":"2","key":"ref30","doi-asserted-by":"crossref","first-page":"314","DOI":"10.1016\/j.nbd.2005.03.012","article-title":"Progressively reduced synaptic vesicle pool size in cultured neurons derived from neuronal ceroid lipofuscinosis-1 knockout mice","volume":"20","author":"T Virmani","year":"2005","journal-title":"Neurobiol Dis"},{"issue":"2","key":"ref31","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1006\/mcne.2001.1010","article-title":"Neuronal trafficking of palmitoyl protein thioesterase provides an excellent model to study the effects of different mutations which cause infantile neuronal ceroid lipofuscinocis","volume":"18","author":"T Salonen","year":"2001","journal-title":"Mol Cell Neurosci"},{"issue":"1","key":"ref32","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1093\/hmg\/10.1.69","article-title":"Palmitoyl protein thioesterase (PPT) localizes into synaptosomes and synaptic vesicles in neurons: implications for infantile neuronal ceroid lipofuscinosis (INCL)","volume":"10","author":"M Lehtovirta","year":"2001","journal-title":"Hum Mol Genet"},{"issue":"2","key":"ref33","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1006\/mgme.2000.2961","article-title":"Expression of palmitoyl protein thioesterase in neurons","volume":"69","author":"O Heinonen","year":"2000","journal-title":"Mol Genet Metab"},{"issue":"9","key":"ref34","doi-asserted-by":"crossref","first-page":"3075","DOI":"10.1172\/JCI33482","article-title":"Palmitoyl protein thioesterase-1 deficiency impairs synaptic vesicle recycling at nerve terminals, contributing to neuropathology in humans and mice","volume":"118","author":"SJ Kim","year":"2008","journal-title":"J Clin Invest"},{"issue":"11","key":"ref35","doi-asserted-by":"crossref","first-page":"1906","DOI":"10.1016\/j.bbadis.2013.05.026","article-title":"Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease)","volume":"1832","author":"JA Hawkins-Salsbury","year":"2013","journal-title":"Biochimica et biophysica acta"},{"issue":"6","key":"ref36","doi-asserted-by":"crossref","first-page":"867","DOI":"10.2174\/138920111795542633","article-title":"Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses","volume":"12","author":"R Kohan","year":"2011","journal-title":"Current pharmaceutical biotechnology"},{"issue":"4","key":"ref37","doi-asserted-by":"crossref","first-page":"374","DOI":"10.1016\/j.ymgme.2009.12.002","article-title":"Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis","volume":"99","author":"JY Lu","year":"2010","journal-title":"Molecular genetics and metabolism"},{"issue":"1\u20132","key":"ref38","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.ymgme.2012.05.009","article-title":"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","volume":"107","author":"J Hu","year":"2012","journal-title":"Molecular genetics and metabolism"},{"key":"ref39","article-title":"Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis","author":"JY Lu","year":"2015","journal-title":"Molecular genetics and metabolism"},{"issue":"3","key":"ref40","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1016\/j.ymgme.2011.05.021","article-title":"Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients","volume":"104","author":"C Sarkar","year":"2011","journal-title":"Molecular genetics and metabolism"},{"issue":"9","key":"ref41","doi-asserted-by":"crossref","first-page":"1159","DOI":"10.1177\/0883073813494480","article-title":"Treatment of the Ppt1(-\/-) mouse model of infantile neuronal ceroid lipofuscinosis with the N-methyl-D-aspartate (NMDA) receptor antagonist memantine","volume":"28","author":"R Finn","year":"2013","journal-title":"Journal of child neurology"},{"key":"ref42","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1007\/8904_2013_226","article-title":"Substrate Reduction Therapy in Four Patients with Milder CLN1 Mutations and Juvenile-Onset Batten Disease Using Cysteamine Bitartrate","volume":"11","author":"M Gavin","year":"2013","journal-title":"JIMD reports"},{"issue":"11","key":"ref43","doi-asserted-by":"crossref","first-page":"1608","DOI":"10.1038\/nn.3526","article-title":"Neuroprotection and lifespan extension in Ppt1(-\/-) mice by NtBuHA: therapeutic implications for INCL","volume":"16","author":"C Sarkar","year":"2013","journal-title":"Nature neuroscience"},{"issue":"6","key":"ref44","doi-asserted-by":"crossref","first-page":"797","DOI":"10.1002\/ana.23545","article-title":"Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis","volume":"71","author":"SL Macauley","year":"2012","journal-title":"Annals of neurology"},{"issue":"5","key":"ref45","doi-asserted-by":"crossref","first-page":"847","DOI":"10.1007\/s10545-011-9446-x","article-title":"Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis","volume":"35","author":"MS Roberts","year":"2012","journal-title":"Journal of inherited metabolic disease"},{"issue":"7469","key":"ref46","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1038\/nature12587","article-title":"Deterministic direct reprogramming of somatic cells to pluripotency","volume":"502","author":"Y Rais","year":"2013","journal-title":"Nature"},{"issue":"5","key":"ref47","doi-asserted-by":"crossref","first-page":"872","DOI":"10.1016\/j.cell.2010.04.010","article-title":"Derivation of pre-X inactivation human embryonic stem cells under physiological oxygen concentrations","volume":"141","author":"CJ Lengner","year":"2010","journal-title":"Cell"},{"issue":"6121","key":"ref48","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex genome engineering using CRISPR\/Cas systems","volume":"339","author":"L Cong","year":"2013","journal-title":"Science"},{"issue":"6121","key":"ref49","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1126\/science.1232033","article-title":"RNA-guided human genome engineering via Cas9","volume":"339","author":"P Mali","year":"2013","journal-title":"Science"},{"issue":"6166","key":"ref50","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1126\/science.1247005","article-title":"Genome-scale CRISPR-Cas9 knockout screening in human cells","volume":"343","author":"O Shalem","year":"2014","journal-title":"Science"},{"key":"ref51","article-title":"Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects","author":"B Shen","year":"2014","journal-title":"Nat Methods"},{"issue":"11","key":"ref52","doi-asserted-by":"crossref","first-page":"2281","DOI":"10.1038\/nprot.2013.143","article-title":"Genome engineering using the CRISPR-Cas9 system","volume":"8","author":"FA Ran","year":"2013","journal-title":"Nat Protoc"},{"issue":"13","key":"ref53","doi-asserted-by":"crossref","first-page":"4967","DOI":"10.1021\/ja810122f","article-title":"Robust fluorescent detection of protein fatty-acylation with chemical reporters","volume":"131","author":"G Charron","year":"2009","journal-title":"J Am Chem Soc"},{"issue":"4","key":"ref54","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1006\/mgme.1999.2809","article-title":"A rapid fluorogenic palmitoyl-protein thioesterase assay: pre- and postnatal diagnosis of INCL","volume":"66","author":"OP van Diggelen","year":"1999","journal-title":"Mol Genet Metab"},{"issue":"11","key":"ref55","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1093\/protein\/gzn039","article-title":"CSS-Palm 2.0: an updated software for palmitoylation sites prediction","volume":"21","author":"J Ren","year":"2008","journal-title":"Protein engineering, design & selection: PEDS"},{"issue":"49","key":"ref56","doi-asserted-by":"crossref","first-page":"12758","DOI":"10.1523\/JNEUROSCI.4214-06.2006","article-title":"GODZ-mediated palmitoylation of GABA(A) receptors is required for normal assembly and function of GABAergic inhibitory synapses","volume":"26","author":"C Fang","year":"2006","journal-title":"J Neurosci"},{"issue":"8","key":"ref57","doi-asserted-by":"crossref","first-page":"2605","DOI":"10.1096\/fj.08-127399","article-title":"Neuronal palmitoyl acyl transferases exhibit distinct substrate specificity","volume":"23","author":"K Huang","year":"2009","journal-title":"FASEB J"},{"issue":"4","key":"ref58","doi-asserted-by":"crossref","first-page":"474","DOI":"10.1016\/j.bbalip.2006.03.010","article-title":"Intracellular localization and tissue-specific distribution of human and yeast DHHC cysteine-rich domain-containing proteins","volume":"1761","author":"Y Ohno","year":"2006","journal-title":"Biochim Biophys Acta"},{"issue":"3","key":"ref59","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1006\/geno.1996.0292","article-title":"cDNA and genomic cloning of human palmitoyl-protein thioesterase (PPT), the enzyme defective in infantile neuronal ceroid lipofuscinosis","volume":"34","author":"JE Schriner","year":"1996","journal-title":"Genomics"},{"issue":"26","key":"ref60","doi-asserted-by":"crossref","first-page":"5881","DOI":"10.1523\/JNEUROSCI.1037-04.2004","article-title":"The gamma2 subunit of GABA(A) receptors is a substrate for palmitoylation by GODZ","volume":"24","author":"CA Keller","year":"2004","journal-title":"J Neurosci"},{"issue":"1","key":"ref61","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1083\/jcb.200903101","article-title":"Mobile DHHC palmitoylating enzyme mediates activity-sensitive synaptic targeting of PSD-95","volume":"186","author":"J Noritake","year":"2009","journal-title":"J Cell Biol"},{"issue":"11","key":"ref62","doi-asserted-by":"crossref","first-page":"1930","DOI":"10.1091\/mbc.E10-10-0824","article-title":"Subcellular Golgi localization of stathmin family proteins is promoted by a specific set of DHHC palmitoyl transferases","volume":"22","author":"AD Levy","year":"2011","journal-title":"Mol Biol Cell"},{"issue":"10","key":"ref63","doi-asserted-by":"crossref","first-page":"4869","DOI":"10.1016\/S0021-9258(18)68866-0","article-title":"Mammalian protein secretion without signal peptide removal. Biosynthesis of plasminogen activator inhibitor-2 in U-937 cells","volume":"263","author":"RD Ye","year":"1988","journal-title":"J Biol Chem"},{"issue":"5","key":"ref64","doi-asserted-by":"crossref","first-page":"1503","DOI":"10.1002\/j.1460-2075.1990.tb08268.x","article-title":"A novel secretory pathway for interleukin-1 beta, a protein lacking a signal sequence","volume":"9","author":"A Rubartelli","year":"1990","journal-title":"EMBO J"},{"issue":"3","key":"ref65","doi-asserted-by":"crossref","first-page":"86","DOI":"10.1016\/0968-0004(90)90186-F","article-title":"A novel pathway for secretory proteins?","volume":"15","author":"A Muesch","year":"1990","journal-title":"Trends Biochem Sci"},{"issue":"4","key":"ref66","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1093\/protein\/gzh037","article-title":"Feature-based prediction of non-classical and leaderless protein secretion","volume":"17","author":"JD Bendtsen","year":"2004","journal-title":"Protein engineering, design & selection: PEDS"},{"issue":"9","key":"ref67","doi-asserted-by":"crossref","first-page":"6498","DOI":"10.1016\/S0021-9258(17)37399-4","article-title":"Regulation of enzymatic activity by active site fatty acylation. A new role for long chain fatty acid acylation of proteins","volume":"269","author":"L Berthiaume","year":"1994","journal-title":"J Biol Chem"},{"issue":"49","key":"ref68","doi-asserted-by":"crossref","first-page":"45704","DOI":"10.1074\/jbc.M102766200","article-title":"Regulation of mitochondrial carbamoyl-phosphate synthetase 1 activity by active site fatty acylation","volume":"276","author":"MM Corvi","year":"2001","journal-title":"J Biol Chem"},{"key":"ref69","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1146\/annurev.biochem.73.011303.073954","article-title":"Palmitoylation of intracellular signaling proteins: regulation and function","volume":"73","author":"JE Smotrys","year":"2004","journal-title":"Annu Rev Biochem"},{"issue":"3","key":"ref70","doi-asserted-by":"crossref","first-page":"538","DOI":"10.1016\/j.ymthe.2005.11.008","article-title":"CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis","volume":"13","author":"MA Griffey","year":"2006","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"issue":"3","key":"ref71","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1016\/j.ymthe.2005.04.018","article-title":"AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis","volume":"12","author":"M Griffey","year":"2005","journal-title":"Molecular therapy: the journal of the American Society of Gene Therapy"},{"issue":"2","key":"ref72","doi-asserted-by":"crossref","first-page":"360","DOI":"10.1016\/j.nbd.2004.03.005","article-title":"Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis","volume":"16","author":"M Griffey","year":"2004","journal-title":"Neurobiology of disease"},{"issue":"1","key":"ref73","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1016\/j.bbrc.2010.03.137","article-title":"Palmitoyl:protein thioesterase (PPT1) inhibitors can act as pharmacological chaperones in infantile Batten disease","volume":"395","author":"G Dawson","year":"2010","journal-title":"Biochemical and biophysical research communications"},{"key":"ref74","article-title":"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","author":"J Hu","year":"2012","journal-title":"Mol Genet Metab"}],"container-title":["PLOS ONE"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/dx.plos.org\/10.1371\/journal.pone.0146466","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,5,31]],"date-time":"2022-05-31T23:11:49Z","timestamp":1654038709000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0146466"}},"subtitle":[],"editor":[{"given":"Anthony Robert","family":"White","sequence":"first","affiliation":[],"role":[{"role":"editor","vocabulary":"crossref"}]}],"short-title":[],"issued":{"date-parts":[[2016,1,5]]},"references-count":74,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2016,1,5]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0146466","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,1,5]]}}}