DOI: 10.1038/mt.2008.73. Systemic AAV6 Delivery Mediating RNA Interference Against SOD1: Neuromuscular Transduction Does Not Alter Disease Progression in fALS Mice

Systemic AAV6 Delivery Mediating RNA Interference Against SOD1: Neuromuscular Transduction Does Not Alter Disease Progression in fALS Mice

10.1038/mt.2008.73

Crossref journal-article

Elsevier BV

Molecular Therapy (78)

Bibliography

Towne, C., Raoul, C., Schneider, B. L., & Aebischer, P. (2008). Systemic AAV6 Delivery Mediating RNA Interference Against SOD1: Neuromuscular Transduction Does Not Alter Disease Progression in fALS Mice. Molecular Therapy, 16(6), 1018â1025.

Authors 4

Chris Towne (first)
Cédric Raoul (additional)
Bernard L Schneider (additional)
Patrick Aebischer (additional)

References 33 Referenced 102

10.1146/annurev.neuro.27.070203.144244 / Annu Rev Neurosci / Unravelling the mechanisms involved in motor neuron degeneration in ALS by Bruijn (2004)
10.1074/jbc.M507685200 / J Biol Chem / Transgenic small interfering RNA halts amyotrophics lateral sclerosis in a mouse model by Saito (2005)
10.1172/JCI25424 / J Clin Invest / Antisense oligonucleotide therapy for neurodegeneratice disease by Smith (2006)
10.1038/nm1207 / Nat Med / Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS by Raoul (2005)
10.1038/nm1205 / Nat Med / Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model by Ralph (2005)
10.1016/j.ymthe.2005.11.009 / Mol Ther / Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders by Mandel (2006)
10.1161/01.RES.0000237661.18885.f6 / Circ Res / Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo by Pacak (2006)
10.1016/j.ymthe.2005.01.012 / Mol Ther / Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter by Sun (2005)
10.1038/sj.gt.3302961 / Gene Ther / Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice by Fu (2007)
10.1038/sj.gt.3302684 / Gene Ther / Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer by Ding (2006)
10.1038/nm1085 / Nat Med / Systemic delivery of genes to striated muscles using adeno-associated viral vectors by Gregorevic (2004)
10.1038/sj.gt.3302142 / Gene Ther / Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B by Harding (2004)
10.2337/db06-0495 / Diabetes / Systemic expression of heme oxygenase-1 ameliorates type 1 diabetes in NOD mice by Hu (2007)
10.1126/science.1086137 / Science / Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model by Kaspar (2003)
10.1002/jgm.556 / J Gene Med / Optimization of an siRNA-expression system with an improved hairpin and its significant suppressive effects in mammalian cells by Miyagishi (2004)
10.1128/JVI.72.1.180-190.1998 / J Virol / Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: involvement of NFkappaB by Löser (1998)
10.1016/j.nbd.2003.12.016 / Neurobiol Dis / The blood-brain barrier: an overview: structure, regulation, and clinical implications by Ballabh (2004)
10.1016/j.brainres.2007.04.044 / Brain Res / Ultrastructure of blood-brain barrier and blood-spinal cord barrier in SOD1 mice modeling ALS by Garbuzova-Davis (2007)
10.1523/JNEUROSCI.20-02-00660.2000 / J Neurosci / Restricted expression of G86R Cu/Zn superoxide dismutase in astrocytes results in astrocytosis but does not cause motoneuron degeneration by Gong (2000)
10.1523/JNEUROSCI.21-10-03369.2001 / J Neurosci / Neuron-specific expression of mutant superoxide dismutase 1 in transgenic mice does not lead to motor impairment by Pramatarova (2001)
10.1523/JNEUROSCI.22-12-04825.2002 / J Neurosci / Accumulation of SOD1 mutants in postnatal motoneurons does not cause motoneuron pathology or motoneuron disease by Lino (2002)
10.1126/science.1086071 / Science / Wild-type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice by Clement (2003)
10.1016/j.neuron.2006.09.018 / Neuron / ALS: a disease of motor neurons and their nonneuronal neighbors by Boillée (2006)
10.1038/nn1876 / Nat Neurosci / Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons by Nagai (2007)
10.1016/j.expneurol.2003.10.004 / Exp Neurol / Amyotrophic lateral sclerosis is a distal axonopathy: evidence in mice and man by Fischer (2004)
10.1096/fj.02-1182fje / FASEB J / Up-regulation of mitochondrial uncoupling protein 3 reveals an early muscular metabolic defect in amyotrophic lateral sclerosis by Dupuis (2003)
10.1016/S0960-8966(01)00240-1 / Neuromuscul Disord / Selective changes in mitochondria respiratory properties in oxidative or glycolytic muscle fibers isolated from G93AhumanSOD1 transgenic mice by Leclerc (2001)
10.1016/j.molbrainres.2004.07.002 / Brain Res Mol Brain Res / Effect of transgene copy number on survival in the G93A SOD1 transgenic mouse model of ALS by Alexander (2004)
10.1073/pnas.0609411103 / Proc Natl Acad Sci USA / Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis by Miller (2006)
10.1016/j.virol.2005.06.051 / Virology / A combination of mutations enhances the neurotropism of AAV-2 by Xu (2005)
10.1089/104303402320987888 / Hum Gene Ther / Lentiviral-mediated RNA interference by Abbas-Terki (2002)
10.1016/S1525-0016(03)00095-9 / Mol Ther / Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6 by Grimm (2003)
10.1016/j.nbd.2004.01.017 / Neurobiol Dis / Local GDNF expression mediated by lentiviral vector protects facial nerve motoneurons but not spinal motoneurons in SOD1(G93A) transgenic mice by Guillot (2004)

Dates

Type	When
Created	17 years, 4 months ago (April 15, 2008, 12:15 p.m.)
Deposited	5 years, 3 months ago (May 4, 2020, 6:19 a.m.)
Indexed	5 months, 1 week ago (March 22, 2025, 5:06 a.m.)
Issued	17 years, 2 months ago (June 1, 2008)
Published	17 years, 2 months ago (June 1, 2008)
Published Print	17 years, 2 months ago (June 1, 2008)

Funders 0

None

BibTeX

@article{Towne_2008, title={Systemic AAV6 Delivery Mediating RNA Interference Against SOD1: Neuromuscular Transduction Does Not Alter Disease Progression in fALS Mice}, volume={16}, ISSN={1525-0016}, url={http://dx.doi.org/10.1038/mt.2008.73}, DOI={10.1038/mt.2008.73}, number={6}, journal={Molecular Therapy}, publisher={Elsevier BV}, author={Towne, Chris and Raoul, Cédric and Schneider, Bernard L and Aebischer, Patrick}, year={2008}, month=jun, pages={1018–1025} }

JSON

{
  "indexed": {
    "date-parts": [
      [
        2025,
        3,
        22
      ]
    ],
    "date-time": "2025-03-22T09:06:24Z",
    "timestamp": 1742634384788
  },
  "reference-count": 33,
  "publisher": "Elsevier BV",
  "issue": "6",
  "license": [
    {
      "start": {
        "date-parts": [
          [
            2008,
            6,
            1
          ]
        ],
        "date-time": "2008-06-01T00:00:00Z",
        "timestamp": 1212278400000
      },
      "content-version": "tdm",
      "delay-in-days": 0,
      "URL": "https://www.elsevier.com/tdm/userlicense/1.0/"
    },
    {
      "start": {
        "date-parts": [
          [
            2016,
            10,
            7
          ]
        ],
        "date-time": "2016-10-07T00:00:00Z",
        "timestamp": 1475798400000
      },
      "content-version": "vor",
      "delay-in-days": 3050,
      "URL": "http://creativecommons.org/licenses/by-nc-nd/4.0/"
    }
  ],
  "content-domain": {
    "domain": [],
    "crossmark-restriction": false
  },
  "published-print": {
    "date-parts": [
      [
        2008,
        6
      ]
    ]
  },
  "DOI": "10.1038/mt.2008.73",
  "type": "journal-article",
  "created": {
    "date-parts": [
      [
        2008,
        4,
        15
      ]
    ],
    "date-time": "2008-04-15T16:15:57Z",
    "timestamp": 1208276157000
  },
  "page": "1018-1025",
  "source": "Crossref",
  "is-referenced-by-count": 102,
  "title": "Systemic AAV6 Delivery Mediating RNA Interference Against SOD1: Neuromuscular Transduction Does Not Alter Disease Progression in fALS Mice",
  "prefix": "10.1016",
  "volume": "16",
  "author": [
    {
      "given": "Chris",
      "family": "Towne",
      "sequence": "first",
      "affiliation": []
    },
    {
      "given": "C\u00e9dric",
      "family": "Raoul",
      "sequence": "additional",
      "affiliation": []
    },
    {
      "given": "Bernard L",
      "family": "Schneider",
      "sequence": "additional",
      "affiliation": []
    },
    {
      "given": "Patrick",
      "family": "Aebischer",
      "sequence": "additional",
      "affiliation": []
    }
  ],
  "member": "78",
  "reference": [
    {
      "key": "10.1038/mt.2008.73_bib1",
      "doi-asserted-by": "crossref",
      "first-page": "723",
      "DOI": "10.1146/annurev.neuro.27.070203.144244",
      "article-title": "Unravelling the mechanisms involved in motor neuron degeneration in ALS",
      "volume": "27",
      "author": "Bruijn",
      "year": "2004",
      "journal-title": "Annu Rev Neurosci"
    },
    {
      "key": "10.1038/mt.2008.73_bib2",
      "doi-asserted-by": "crossref",
      "first-page": "42826",
      "DOI": "10.1074/jbc.M507685200",
      "article-title": "Transgenic small interfering RNA halts amyotrophics lateral sclerosis in a mouse model",
      "volume": "280",
      "author": "Saito",
      "year": "2005",
      "journal-title": "J Biol Chem"
    },
    {
      "key": "10.1038/mt.2008.73_bib3",
      "doi-asserted-by": "crossref",
      "first-page": "2290",
      "DOI": "10.1172/JCI25424",
      "article-title": "Antisense oligonucleotide therapy for neurodegeneratice disease",
      "volume": "116",
      "author": "Smith",
      "year": "2006",
      "journal-title": "J Clin Invest"
    },
    {
      "key": "10.1038/mt.2008.73_bib4",
      "doi-asserted-by": "crossref",
      "first-page": "423",
      "DOI": "10.1038/nm1207",
      "article-title": "Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS",
      "volume": "11",
      "author": "Raoul",
      "year": "2005",
      "journal-title": "Nat Med"
    },
    {
      "key": "10.1038/mt.2008.73_bib5",
      "doi-asserted-by": "crossref",
      "first-page": "429",
      "DOI": "10.1038/nm1205",
      "article-title": "Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model",
      "volume": "11",
      "author": "Ralph",
      "year": "2005",
      "journal-title": "Nat Med"
    },
    {
      "key": "10.1038/mt.2008.73_bib6",
      "doi-asserted-by": "crossref",
      "first-page": "463",
      "DOI": "10.1016/j.ymthe.2005.11.009",
      "article-title": "Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders",
      "volume": "13",
      "author": "Mandel",
      "year": "2006",
      "journal-title": "Mol Ther"
    },
    {
      "key": "10.1038/mt.2008.73_bib7",
      "doi-asserted-by": "crossref",
      "first-page": "e3",
      "DOI": "10.1161/01.RES.0000237661.18885.f6",
      "article-title": "Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo",
      "volume": "99",
      "author": "Pacak",
      "year": "2006",
      "journal-title": "Circ Res"
    },
    {
      "key": "10.1038/mt.2008.73_bib8",
      "doi-asserted-by": "crossref",
      "first-page": "889",
      "DOI": "10.1016/j.ymthe.2005.01.012",
      "article-title": "Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter",
      "volume": "11",
      "author": "Sun",
      "year": "2005",
      "journal-title": "Mol Ther"
    },
    {
      "key": "10.1038/mt.2008.73_bib9",
      "doi-asserted-by": "crossref",
      "first-page": "1065",
      "DOI": "10.1038/sj.gt.3302961",
      "article-title": "Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice",
      "volume": "14",
      "author": "Fu",
      "year": "2007",
      "journal-title": "Gene Ther"
    },
    {
      "key": "10.1038/mt.2008.73_bib10",
      "doi-asserted-by": "crossref",
      "first-page": "587",
      "DOI": "10.1038/sj.gt.3302684",
      "article-title": "Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer",
      "volume": "13",
      "author": "Ding",
      "year": "2006",
      "journal-title": "Gene Ther"
    },
    {
      "key": "10.1038/mt.2008.73_bib11",
      "doi-asserted-by": "crossref",
      "first-page": "828",
      "DOI": "10.1038/nm1085",
      "article-title": "Systemic delivery of genes to striated muscles using adeno-associated viral vectors",
      "volume": "10",
      "author": "Gregorevic",
      "year": "2004",
      "journal-title": "Nat Med"
    },
    {
      "key": "10.1038/mt.2008.73_bib12",
      "doi-asserted-by": "crossref",
      "first-page": "204",
      "DOI": "10.1038/sj.gt.3302142",
      "article-title": "Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B",
      "volume": "11",
      "author": "Harding",
      "year": "2004",
      "journal-title": "Gene Ther"
    },
    {
      "key": "10.1038/mt.2008.73_bib13",
      "doi-asserted-by": "crossref",
      "first-page": "1240",
      "DOI": "10.2337/db06-0495",
      "article-title": "Systemic expression of heme oxygenase-1 ameliorates type 1 diabetes in NOD mice",
      "volume": "56",
      "author": "Hu",
      "year": "2007",
      "journal-title": "Diabetes"
    },
    {
      "key": "10.1038/mt.2008.73_bib14",
      "doi-asserted-by": "crossref",
      "first-page": "839",
      "DOI": "10.1126/science.1086137",
      "article-title": "Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model",
      "volume": "301",
      "author": "Kaspar",
      "year": "2003",
      "journal-title": "Science"
    },
    {
      "key": "10.1038/mt.2008.73_bib15",
      "doi-asserted-by": "crossref",
      "first-page": "715",
      "DOI": "10.1002/jgm.556",
      "article-title": "Optimization of an siRNA-expression system with an improved hairpin and its significant suppressive effects in mammalian cells",
      "volume": "6",
      "author": "Miyagishi",
      "year": "2004",
      "journal-title": "J Gene Med"
    },
    {
      "key": "10.1038/mt.2008.73_bib16",
      "doi-asserted-by": "crossref",
      "first-page": "180",
      "DOI": "10.1128/JVI.72.1.180-190.1998",
      "article-title": "Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: involvement of NFkappaB",
      "volume": "72",
      "author": "L\u00f6ser",
      "year": "1998",
      "journal-title": "J Virol"
    },
    {
      "key": "10.1038/mt.2008.73_bib17",
      "doi-asserted-by": "crossref",
      "first-page": "1",
      "DOI": "10.1016/j.nbd.2003.12.016",
      "article-title": "The blood-brain barrier: an overview: structure, regulation, and clinical implications",
      "volume": "16",
      "author": "Ballabh",
      "year": "2004",
      "journal-title": "Neurobiol Dis"
    },
    {
      "key": "10.1038/mt.2008.73_bib18",
      "doi-asserted-by": "crossref",
      "first-page": "126",
      "DOI": "10.1016/j.brainres.2007.04.044",
      "article-title": "Ultrastructure of blood-brain barrier and blood-spinal cord barrier in SOD1 mice modeling ALS",
      "volume": "1157",
      "author": "Garbuzova-Davis",
      "year": "2007",
      "journal-title": "Brain Res"
    },
    {
      "key": "10.1038/mt.2008.73_bib19",
      "doi-asserted-by": "crossref",
      "first-page": "660",
      "DOI": "10.1523/JNEUROSCI.20-02-00660.2000",
      "article-title": "Restricted expression of G86R Cu/Zn superoxide dismutase in astrocytes results in astrocytosis but does not cause motoneuron degeneration",
      "volume": "20",
      "author": "Gong",
      "year": "2000",
      "journal-title": "J Neurosci"
    },
    {
      "key": "10.1038/mt.2008.73_bib20",
      "doi-asserted-by": "crossref",
      "first-page": "3369",
      "DOI": "10.1523/JNEUROSCI.21-10-03369.2001",
      "article-title": "Neuron-specific expression of mutant superoxide dismutase 1 in transgenic mice does not lead to motor impairment",
      "volume": "21",
      "author": "Pramatarova",
      "year": "2001",
      "journal-title": "J Neurosci"
    },
    {
      "key": "10.1038/mt.2008.73_bib21",
      "doi-asserted-by": "crossref",
      "first-page": "4825",
      "DOI": "10.1523/JNEUROSCI.22-12-04825.2002",
      "article-title": "Accumulation of SOD1 mutants in postnatal motoneurons does not cause motoneuron pathology or motoneuron disease",
      "volume": "22",
      "author": "Lino",
      "year": "2002",
      "journal-title": "J Neurosci"
    },
    {
      "key": "10.1038/mt.2008.73_bib22",
      "doi-asserted-by": "crossref",
      "first-page": "113",
      "DOI": "10.1126/science.1086071",
      "article-title": "Wild-type nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice",
      "volume": "302",
      "author": "Clement",
      "year": "2003",
      "journal-title": "Science"
    },
    {
      "key": "10.1038/mt.2008.73_bib23",
      "doi-asserted-by": "crossref",
      "first-page": "39",
      "DOI": "10.1016/j.neuron.2006.09.018",
      "article-title": "ALS: a disease of motor neurons and their nonneuronal neighbors",
      "volume": "52",
      "author": "Boill\u00e9e",
      "year": "2006",
      "journal-title": "Neuron"
    },
    {
      "key": "10.1038/mt.2008.73_bib24",
      "doi-asserted-by": "crossref",
      "first-page": "615",
      "DOI": "10.1038/nn1876",
      "article-title": "Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons",
      "volume": "10",
      "author": "Nagai",
      "year": "2007",
      "journal-title": "Nat Neurosci"
    },
    {
      "key": "10.1038/mt.2008.73_bib25",
      "doi-asserted-by": "crossref",
      "first-page": "232",
      "DOI": "10.1016/j.expneurol.2003.10.004",
      "article-title": "Amyotrophic lateral sclerosis is a distal axonopathy: evidence in mice and man",
      "volume": "185",
      "author": "Fischer",
      "year": "2004",
      "journal-title": "Exp Neurol"
    },
    {
      "key": "10.1038/mt.2008.73_bib26",
      "doi-asserted-by": "crossref",
      "first-page": "2091",
      "DOI": "10.1096/fj.02-1182fje",
      "article-title": "Up-regulation of mitochondrial uncoupling protein 3 reveals an early muscular metabolic defect in amyotrophic lateral sclerosis",
      "volume": "17",
      "author": "Dupuis",
      "year": "2003",
      "journal-title": "FASEB J"
    },
    {
      "key": "10.1038/mt.2008.73_bib27",
      "doi-asserted-by": "crossref",
      "first-page": "722",
      "DOI": "10.1016/S0960-8966(01)00240-1",
      "article-title": "Selective changes in mitochondria respiratory properties in oxidative or glycolytic muscle fibers isolated from G93AhumanSOD1 transgenic mice",
      "volume": "11",
      "author": "Leclerc",
      "year": "2001",
      "journal-title": "Neuromuscul Disord"
    },
    {
      "key": "10.1038/mt.2008.73_bib28",
      "doi-asserted-by": "crossref",
      "first-page": "7",
      "DOI": "10.1016/j.molbrainres.2004.07.002",
      "article-title": "Effect of transgene copy number on survival in the G93A SOD1 transgenic mouse model of ALS",
      "volume": "130",
      "author": "Alexander",
      "year": "2004",
      "journal-title": "Brain Res Mol Brain Res"
    },
    {
      "key": "10.1038/mt.2008.73_bib29",
      "doi-asserted-by": "crossref",
      "first-page": "19546",
      "DOI": "10.1073/pnas.0609411103",
      "article-title": "Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis",
      "volume": "103",
      "author": "Miller",
      "year": "2006",
      "journal-title": "Proc Natl Acad Sci USA"
    },
    {
      "key": "10.1038/mt.2008.73_bib30",
      "doi-asserted-by": "crossref",
      "first-page": "203",
      "DOI": "10.1016/j.virol.2005.06.051",
      "article-title": "A combination of mutations enhances the neurotropism of AAV-2",
      "volume": "341",
      "author": "Xu",
      "year": "2005",
      "journal-title": "Virology"
    },
    {
      "key": "10.1038/mt.2008.73_bib31",
      "doi-asserted-by": "crossref",
      "first-page": "2197",
      "DOI": "10.1089/104303402320987888",
      "article-title": "Lentiviral-mediated RNA interference",
      "volume": "13",
      "author": "Abbas-Terki",
      "year": "2002",
      "journal-title": "Hum Gene Ther"
    },
    {
      "key": "10.1038/mt.2008.73_bib32",
      "doi-asserted-by": "crossref",
      "first-page": "839",
      "DOI": "10.1016/S1525-0016(03)00095-9",
      "article-title": "Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6",
      "volume": "7",
      "author": "Grimm",
      "year": "2003",
      "journal-title": "Mol Ther"
    },
    {
      "key": "10.1038/mt.2008.73_bib33",
      "doi-asserted-by": "crossref",
      "first-page": "139",
      "DOI": "10.1016/j.nbd.2004.01.017",
      "article-title": "Local GDNF expression mediated by lentiviral vector protects facial nerve motoneurons but not spinal motoneurons in SOD1(G93A) transgenic mice",
      "volume": "16",
      "author": "Guillot",
      "year": "2004",
      "journal-title": "Neurobiol Dis"
    }
  ],
  "container-title": "Molecular Therapy",
  "original-title": [],
  "language": "en",
  "link": [
    {
      "URL": "https://api.elsevier.com/content/article/PII:S1525001616317257?httpAccept=text/plain",
      "content-type": "text/plain",
      "content-version": "vor",
      "intended-application": "text-mining"
    },
    {
      "URL": "https://api.elsevier.com/content/article/PII:S1525001616317257?httpAccept=text/xml",
      "content-type": "text/xml",
      "content-version": "vor",
      "intended-application": "text-mining"
    }
  ],
  "deposited": {
    "date-parts": [
      [
        2020,
        5,
        4
      ]
    ],
    "date-time": "2020-05-04T10:19:39Z",
    "timestamp": 1588587579000
  },
  "score": 1,
  "resource": {
    "primary": {
      "URL": "https://linkinghub.elsevier.com/retrieve/pii/S1525001616317257"
    }
  },
  "subtitle": [],
  "short-title": [],
  "issued": {
    "date-parts": [
      [
        2008,
        6
      ]
    ]
  },
  "references-count": 33,
  "journal-issue": {
    "issue": "6",
    "published-print": {
      "date-parts": [
        [
          2008,
          6
        ]
      ]
    }
  },
  "alternative-id": [
    "S1525001616317257"
  ],
  "URL": "http://dx.doi.org/10.1038/mt.2008.73",
  "relation": {},
  "ISSN": [
    "1525-0016"
  ],
  "subject": [],
  "container-title-short": "Molecular Therapy",
  "published": {
    "date-parts": [
      [
        2008,
        6
      ]
    ]
  }
}