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Title:           An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs
Subject:         Molecular Therapy: Methods & Clinical Development, 32 (2024) 101354. doi:10.1016/j.omtm.2024.101354
Author:          Vidyullatha Vasireddy
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      Title: An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and i ...
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       Item: 
        Title: Introduction
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       Item: 
        Title: Results
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        Children: 
         Item: 
          Title: SBT101 in mixed glial cell cultures from Abcd1-Null mice
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         Item: 
          Title: SBT101 in Abcd1−/y mice
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         Item: 
          Title: SBT101 in Abcd1−/y/Abcd2−/− double-knockout mice
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         Item: 
          Title: Biodistribution in cynomolgus macaques
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         Item: 
          Title: Safety in cynomolgus macaques
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        Title: Discussion
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       Item: 
        Title: Material and methods
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         Item: 
          Title: Study designs for mouse studies
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         Item: 
          Title: Study designs for NHP studies
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         Item: 
          Title: Treatments for mouse studies
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         Item: 
          Title: Treatments for NHP studies
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         Item: 
          Title: Animals for mouse studies
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         Item: 
          Title: Animals for NHP studies
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         Item: 
          Title: Sample collection and assessments for mouse studies
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         Item: 
          Title: Sample collection and assessments for NHP studies
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         Item: 
          Title: hABCD1 protein expression for mouse studies
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         Item: 
          Title: Total VLCFA and C26:0-lysophophatidylcholine levels
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         Item: 
          Title: Viral genomes and mtDNA copy numbers for mouse studies
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         Item: 
          Title: Vector genome biodistribution for NHP studies
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         Item: 
          Title: hABCD1 mRNA analysis for mouse studies
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         Item: 
          Title: hABCD1 mRNA analysis for NHP studies
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         Item: 
          Title: Immunohistochemistry for mouse studies
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         Item: 
          Title: GFP immunohistochemistry for NHP studies
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         Item: 
          Title: TNF-α levels for mouse studies
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         Item: 
          Title: Grip strength for mouse studies
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         Item: 
          Title: Neutralizing antibodies for NHP studies
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         Item: 
          Title: Serum chemistry for NHP studies
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         Item: 
          Title: Histopathology for NHP studies
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          Title: Ethics
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          Title: Statistical analysis
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        Title: Data and code availability
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        Title: Acknowledgments
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        Title: Author contributions
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        Title: Declaration of interests
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  Checksum: 66fa30a5
   Type: CRC32
  Checksum: 5cb2686b8eeabe5eed4c5a580e65f6fa
   Type: MD5
  Checksum: 01ed1ad0f9363cb7b0978199c09bb0b9c55a0516
   Type: SHA-1
  Checksum: 1953281ff75d148a6983fcf9e364c14c958b7f76e34dc9c1a692475bced89236
   Type: SHA-256