Eugenio Montini
YOU?
Author Swipe
View article: Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2
Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2 Open
View article: Spatiotemporal liver dynamics shape hepatocellular heterogeneity and impact in vivo gene engineering
Spatiotemporal liver dynamics shape hepatocellular heterogeneity and impact in vivo gene engineering Open
View article: Changes of Airway Space and Flow in Patients Treated with Rapid Palatal Expander (RPE): An Observational Pilot Study with Comparison with Non-Treated Patients
Changes of Airway Space and Flow in Patients Treated with Rapid Palatal Expander (RPE): An Observational Pilot Study with Comparison with Non-Treated Patients Open
Background/Objectives. With a rapid palatal expander (RPE) is reported to be effective in increasing the volume of nasal cavities, with a restoration of physiological nasal airflow. The purpose of this retrospective clinical study was to e…
View article: In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking
In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking Open
Lentiviral vector (LV)-mediated ex vivo gene therapy for haematopoietic stem and progenitor cells (HSPCs) has delivered on the promise of a 'one-and-done' treatment for several genetic diseases1. However, ex vivo manipulation an…
View article: Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes
Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes Open
View article: Donor-derived CARCIK-CD19 cells engineered with Sleeping Beauty transposon in acute lymphoblastic leukemia relapsed after allogeneic transplantation
Donor-derived CARCIK-CD19 cells engineered with Sleeping Beauty transposon in acute lymphoblastic leukemia relapsed after allogeneic transplantation Open
View article: Long-term lineage commitment in haematopoietic stem cell gene therapy
Long-term lineage commitment in haematopoietic stem cell gene therapy Open
View article: A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID Open
Hematopoietic stem cell gene therapy (GT) using a γ-retroviral vector (γ-RV) is an effective treatment for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency. Here, we describe a case of GT-related T-cell acute lymphobl…
View article: Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis
Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis Open
In physiological conditions, few circulating hematopoietic stem/progenitor cells (cHSPCs) are present in the peripheral blood, but their contribution to human hematopoiesis remain unsolved. By integrating advanced immunophenotyping, single…
View article: P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*
P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up* Open
Metachromatic leukodystrophy (MLD) is a rare lysosomal storage disorder caused by biallelic pathogenic variants in the arylsulfatase A (ARSA) gene that result in deficiency of the ARSA enzyme. ARSA deficiency leads to accumulation of the u…
View article: Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells
Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells Open
View article: S250: UNVEILING THE BIOLOGICAL ROLE OF PERIPHERAL BLOOD HUMAN CIRCULATING HEMATOPOIETIC STEM AND PROGENITOR CELLS
S250: UNVEILING THE BIOLOGICAL ROLE OF PERIPHERAL BLOOD HUMAN CIRCULATING HEMATOPOIETIC STEM AND PROGENITOR CELLS Open
Topic: 23. Hematopoiesis, stem cells and microenvironment Background: Although most hematopoietic stem/progenitor cells (HSPC) reside in the bone marrow (BM), few circulating HSPC (cHSPC) can be also found in the peripheral blood (PB) at s…
View article: Spatio-temporal dynamics of the liver shapes hepatocytes heterogeneity and impacts in vivo gene transfer and editing
Spatio-temporal dynamics of the liver shapes hepatocytes heterogeneity and impacts in vivo gene transfer and editing Open
The liver is a central organ for metabolism and hepatocytes are the main cell type responsible for most of its functions. Several previous studies investigated the cell types involved in tissue homeostasis and regeneration 1–4 , however th…
View article: Balanced SET levels favor the correct enhancer repertoire during cell fate acquisition
Balanced SET levels favor the correct enhancer repertoire during cell fate acquisition Open
View article: Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy
Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy Open
View article: Longitudinal single-cell profiling of chemotherapy response in acute myeloid leukemia
Longitudinal single-cell profiling of chemotherapy response in acute myeloid leukemia Open
Acute myeloid leukemia may be characterized by a fraction of leukemia stem cells (LSCs) that sustain disease propagation eventually leading to relapse. Yet, the contribution of LSCs to early therapy resistance and AML regeneration remains …
View article: Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci
Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci Open
Adeno-associated Viral vectors (AAVs) have been successful exploited in gene therapy applications for the treatment of several genetic disorders. AAV is considered an episomal vector but it has been shown to integrate within the host-cell …
View article: <i>InCliniGene</i> enables high-throughput and comprehensive <i>in vivo</i> clonal tracking toward clinical genomics data integration
<i>InCliniGene</i> enables high-throughput and comprehensive <i>in vivo</i> clonal tracking toward clinical genomics data integration Open
High-throughput clonal tracking in patients under hematopoietic stem cell gene therapy with integrating vector is instrumental in assessing bio-safety and efficacy. Monitoring the fate of millions of transplanted clones and their progeny a…
View article: ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications
ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications Open
Longitudinal clonal tracking studies based on high-throughput sequencing technologies supported safety and long-term efficacy and unraveled hematopoietic reconstitution in many gene therapy applications with unprecedented resolution. Howev…
View article: Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells Open
View article: Balanced SET levels favor the correct enhancer repertoire during cell fate acquisition
Balanced SET levels favor the correct enhancer repertoire during cell fate acquisition Open
SUMMARY Within the chromatin, distal elements interact with promoters to regulate specific transcriptional programs. Histone acetylation, interfering with the net charges of the nucleosomes, is a key player in this regulation. Here, we rep…
View article: Clonal reconstruction from co-occurrence of vector integration sites accurately quantifies expanding clones in vivo
Clonal reconstruction from co-occurrence of vector integration sites accurately quantifies expanding clones in vivo Open
High transduction rates of viral vectors in gene therapies (GT) and experimental hematopoiesis ensure a high frequency of gene delivery, although multiple integration events can occur in the same cell. Therefore, tracing of integration sit…
View article: Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective Open
View article: Normalization of clonal diversity in gene therapy studies using shape constrained splines
Normalization of clonal diversity in gene therapy studies using shape constrained splines Open
View article: Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy Open
Chronic granulomatous disease (CGD) is a rare inherited disorder due to loss-of-function mutations in genes encoding the NADPH oxidase subunits. Hematopoietic stem and progenitor cell (HSPC) gene therapy (GT) using regulated lentiviral vec…
View article: Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy
Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy Open
Chronic granulomatous disease (CGD) is a rare inherited disorder due to loss-of-function mutations in genes encoding the NADPH oxidase subunits. Hematopoietic stem and progenitor cell (HSPC) gene therapy (GT) using regulated lentiviral vec…
View article: S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY
S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY Open
Background: Transfusion-dependent ß-thalassemia (TDT) is a disorder due to mutations in the gene encoding the ß-globin chain causing a reduced or absent production of haemoglobin A leading to severe anaemia and lifelong transfusion depende…
View article: Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device
Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device Open
View article: Investigating the Barrier Activity of Novel, Human Enhancer-Blocking Chromatin Insulators for Hematopoietic Stem Cell Gene Therapy
Investigating the Barrier Activity of Novel, Human Enhancer-Blocking Chromatin Insulators for Hematopoietic Stem Cell Gene Therapy Open
Despite the unequivocal success of hematopoietic stem and progenitor cell gene therapy, limitations still exist including genotoxicity and variegation/silencing of transgene expression. A class of DNA regulatory elements known as chromatin…
View article: AAV integration in human hepatocytes
AAV integration in human hepatocytes Open