Annalisa Lattanzi

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Publications (10)

• Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
  Annalisa Lattanzi, Joab Camarena, Premanjali Lahiri +20 more
  Science Translational Medicine 2021 DOI: 10.1126/scitranslmed.abf2444
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• Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
  Annalisa Lattanzi, Vasco Meneghini, Giulia Pavani +12 more
  Molecular Therapy 2019 DOI: 10.1016/j.ymthe.2018.10.008
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• Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
  Blood 2018 DOI: 10.1182/blood-2017-10-811505
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• Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System
  Molecular Therapy - Nucleic Acids 2017 DOI: 10.1016/j.omtn.2017.02.004
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• Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy
  Molecular Therapy - Methods and Clinical Development 2016 DOI: 10.1038/mtm.2016.60
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• Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy
  EMBO Molecular Medicine 2016 DOI: 10.15252/emmm.201505850
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• Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy
  Human Molecular Genetics 2014 DOI: 10.1093/hmg/ddu034
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• Dynamic Activity of miR-125b and miR-93 during Murine Neural Stem Cell Differentiation In Vitro and in the Subventricular Zone Neurogenic Niche
  PLoS ONE 2013 DOI: 10.1371/journal.pone.0067411
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• Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models
  Human Molecular Genetics 2010 DOI: 10.1093/hmg/ddq099
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• Specific determination of β-galactocerebrosidase activity via competitive inhibition of β-galactosidase
  Clinical Chemistry 2009 DOI: 10.1373/clinchem.2008.115873
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