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List of works by Angelo Leone Lombardo

A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice

scientific article published on 28 August 2007

An unbiased genome-wide analysis of zinc-finger nuclease specificity

scientific article

DNA damage contributes to neurotoxic inflammation in Aicardi-Goutières syndrome astrocytes

scientific article published on 09 March 2022

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer

scientific article

Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors

scientific article published in January 2002

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

scientific article (publication date: 28 October 2007)

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

scientific article

Genome editing: a tool for research and therapy: targeted genome editing hits the clinic

scientific article published on October 2014

Genomic instability in induced stem cells

scientific article

In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors

scientific article published in June 2002

Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing

scientific article published on September 2016

Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient

scientific article published on 01 December 2002

Leukocytes recruited by tumor-derived HMGB1 sustain peritoneal carcinomatosis

scientific article published on 08 January 2016

Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy

scientific article

Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotency

scientific article

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.

scientific article published in October 2017

Site-specific integration and tailoring of cassette design for sustainable gene transfer.

scientific article published on 21 August 2011

TRPML1 links lysosomal calcium to autophagosome biogenesis through the activation of the CaMKKβ/VPS34 pathway

scientific article published on 10 December 2019

Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts

scientific article

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination

scientific article published on 13 June 2013

Targeted gene therapy and cell reprogramming in Fanconi anemia

scientific article published on 23 May 2014

Targeted genome editing in human repopulating haematopoietic stem cells

scientific article published on 28 May 2014

Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

scientific article

The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity

scientific article

The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4+CD25+ regulatory T cells.

scientific article published on 10 May 2007

iPSC-derived neural precursors exert a neuroprotective role in immune-mediated demyelination via the secretion of LIF.

scientific article published in January 2013

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