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Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

Nature medicine, 2019-02, Vol.25 (2), p.234-241

2019

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ARPIR: automatic RNA-Seq pipelines with interactive report

BMC bioinformatics, 2020-12, Vol.21 (Suppl 19), p.574-574, Article 574

2020

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Retrieval of vector integration sites from cell-free DNA

Nature medicine, 2021-08, Vol.27 (8), p.1458-1470

2021

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VISPA2: a scalable pipeline for high-throughput identification and annotation of vector integration sites

BMC bioinformatics, 2017-11, Vol.18 (1), p.520-520, Article 520

2017

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GATA1 epigenetic deregulation contributes to the development of AML with NPM1 and FLT3-ITD cooperating mutations

Leukemia, 2019-07, Vol.33 (7), p.1827-1832

2019

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SiCoDEA: A Simple, Fast and Complete App for Analyzing the Effect of Individual Drugs and Their Combinations

Biomolecules (Basel, Switzerland), 2022-06, Vol.12 (7), p.904

2022

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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

The New England journal of medicine, 2021-11, Vol.385 (21), p.1929-1940

2021

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ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications

Briefings in bioinformatics, 2023-01, Vol.24 (1)

2023

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adLIMS: a customized open source software that allows bridging clinical and basic molecular research studies

BMC bioinformatics, 2015-06, Vol.16 Suppl 9 (S9), p.S5-S5, Article S5

2015

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HGG-47. SPATIAL SINGLE-CELL PROTEOMIC ANALYSIS MAPS THE DISTINCT PEDIATRIC-TYPE DIFFUSE HIGH-GRADE GLIOMA TUMOR ENTITIES AND DEFINES THEIR TUMOR AND IMMUNE MICROENVIRONMENTS

Neuro-oncology (Charlottesville, Va.), 2024-06, Vol.26 (Suppl 4), p.0-0

2024

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Abstract P195: Omacetaxine mepessucinate plus venetoclax show strong synergistic anti-leukemic activity in nucleophosmin ( NPM1 )-mutated AML patient-derived xenograft (PDX) models to support a phase 1 clinical trial

Molecular cancer therapeutics, 2021-12, Vol.20 (12_Supplement), p.P195-P195

2021

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γ-TRIS: a graph-algorithm for comprehensive identification of vector genomic insertion sites

Bioinformatics, 2020-03, Vol.36 (5), p.1622-1624

2020

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Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings

Human gene therapy, 2019-09, Vol.30 (9), p.1133

2019

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Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice

Blood, 2023-05, Vol.141 (19), p.2316-2329

2023

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Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells

Molecular therapy, 2024-01, Vol.32 (1), p.124-139

2024

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Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies

Human gene therapy, 2016-10, Vol.27 (10), p.813

2016

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Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients

Blood, 2022-11, Vol.140 (Supplement 1), p.1904-1905

2022

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InCliniGene enables high-throughput and comprehensive in vivo clonal tracking toward clinical genomics data integration

Database : the journal of biological databases and curation, 2023-11, Vol.2023

2023

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A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID

Nature communications, 2024-04, Vol.15 (1), p.3662-18, Article 3662

2024

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212. Lentiviral Insertional Mutagenesis Helps to Uncover the Mechanisms of Resistance to AZD9291 and CO-1686 in EGFR-Mutant Lung Adenocarcinoma

Molecular therapy, 2016-05, Vol.24, p.S83-S83

2016

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529. Lentiviral Vectors with a Reduced Splicing Interference Potential Have a Significantly Improved Safety Profile In Vivo

Molecular therapy, 2016-05, Vol.24, p.S211-S212

2016

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Prolonged XPO1 inhibition is essential for optimal antileukemic activity in NPM1-mutated AML

Blood advances, 2022-11, Vol.6 (22), p.5938-5949

2022

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Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

ARPIR: automatic RNA-Seq pipelines with interactive report

Retrieval of vector integration sites from cell-free DNA

VISPA2: a scalable pipeline for high-throughput identification and annotation of vector integration sites

Dactinomycin induces complete remission associated with nucleolar stress response in relapsed/refractory NPM1-mutated AML

GATA1 epigenetic deregulation contributes to the development of AML with NPM1 and FLT3-ITD cooperating mutations

SiCoDEA: A Simple, Fast and Complete App for Analyzing the Effect of Individual Drugs and Their Combinations

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications

adLIMS: a customized open source software that allows bridging clinical and basic molecular research studies

Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci

HGG-47. SPATIAL SINGLE-CELL PROTEOMIC ANALYSIS MAPS THE DISTINCT PEDIATRIC-TYPE DIFFUSE HIGH-GRADE GLIOMA TUMOR ENTITIES AND DEFINES THEIR TUMOR AND IMMUNE MICROENVIRONMENTS

Abstract P195: Omacetaxine mepessucinate plus venetoclax show strong synergistic anti-leukemic activity in nucleophosmin ( NPM1 )-mutated AML patient-derived xenograft (PDX) models to support a phase 1 clinical trial

γ-TRIS: a graph-algorithm for comprehensive identification of vector genomic insertion sites

Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Ex Vivo Gene Therapy Settings

Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice

Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells

Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis

Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies

Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients

InCliniGene enables high-throughput and comprehensive in vivo clonal tracking toward clinical genomics data integration

A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID

212. Lentiviral Insertional Mutagenesis Helps to Uncover the Mechanisms of Resistance to AZD9291 and CO-1686 in EGFR-Mutant Lung Adenocarcinoma

529. Lentiviral Vectors with a Reduced Splicing Interference Potential Have a Significantly Improved Safety Profile In Vivo

Prolonged XPO1 inhibition is essential for optimal antileukemic activity in NPM1-mutated AML

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