Luca Biasco
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View article: Mortality and Neurodevelopmental Outcome in an Italian Cohort of Very Low Birth Weight Infants
Mortality and Neurodevelopmental Outcome in an Italian Cohort of Very Low Birth Weight Infants Open
Aim Preterm infants face high risks of mortality and neurodevelopmental impairment. We aimed to evaluate the outcomes in an Italian cohort of very low birth weight infants. Methods This multicenter prospective study included very low birth…
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article Open
Regulators and industry are actively seeking improvements and alternatives to current models and approaches to evaluate potential carcinogenicity of gene therapies (GTs). A meeting of invited experts was organized by NC3Rs/UKEMS (London, M…
Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations Open
Pompe disease, a rare genetic neuromuscular disorder, is caused by a deficiency of acid alpha-glucosidase (GAA), leading to the accumulation of glycogen in lysosomes and the progressive development of muscle weakness. The current standard …
View article: Efficient and Specific <i>In Vivo</i> Genetic Engineering of Human Hematopoietic Stem Progenitor Cells without Selective Conditioning
Efficient and Specific <i>In Vivo</i> Genetic Engineering of Human Hematopoietic Stem Progenitor Cells without Selective Conditioning Open
Achieving in vivo genetic engineering of hematopoietic stem progenitor cells (HSPC) has the potential to transform treatment for hematological disorders. An ideal in vivo delivery platform should provide access to resting HSPC at low doses…
IS-Seq: a bioinformatics pipeline for integration sites analysis with comprehensive abundance quantification methods Open
Background Integration site (IS) analysis is a fundamental analytical platform for evaluating the safety and efficacy of viral vector based preclinical and clinical Gene Therapy (GT). A handful of groups have developed standardized bioinfo…
IS-Seq: a bioinformatics pipeline for integration sites analysis with comprehensive abundance quantification methods Open
Integration site (IS) analysis is a fundamental analytical platform for evaluating the safety and efficacy of viral vector based preclinical and clinical Gene Therapy (GT). A handful of groups have developed standardized bioinformatics pip…
Additional file 3 of IS-Seq: a bioinformatics pipeline for integration sites analysis with comprehensive abundance quantification methods Open
Additional file 3: Raw data relative to Tables 1-5, Figures 4-7, Supplementary Table S9 and Supplementary Figures S2, S4-S6.
TRACKING HEMATOPOIETIC STEM CELL EVOLUTION IN A WISKOTT-ALDRICH CLINICAL TRIAL Open
Hematopoietic Stem Cells (HSC) are the cells that give rise to all other blood cells and, as such, they are crucial in the healthy development of individuals. Wiskott-Aldrich Syndrome (WAS) is a severe disorder affecting the regulation of …
High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing Open
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) has shown clear neurological benefit in rare diseases, which is achieved through the engraftment of genetically modified microglia-like cells (MLCs) in the brain. Still, the engraft…
Genetically engineered microglia-like cells have therapeutic potential for neurodegenerative disease Open
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) results in the engraftment of genetically modified microglia-like cells (MLCs) in the brain. While HSPC-GT has shown a clear neurological benefit in the clinic for specific rare dis…
Penalized inference of the hematopoietic cell differentiation network via high-dimensional clonal tracking Open
Background During their lifespan, stem- or progenitor cells have the ability to differentiate into more committed cell lineages. Understanding this process can be key in treating certain diseases. However, up until now only limited informa…
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study Open
Italian Telethon Foundation, GlaxoSmithKline, and Orchard Therapeutics.
View article: Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients
Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients Open
Novel therapies for sickle cell disease (SCD) based on genetically engineered autologous hematopoietic stem and progenitor cells (HSPCs) are critically dependent on a safe and effective strategy for cell procurement. We sought to assess th…
CRISPR-SURF: Discovering regulatory elements by deconvolution of CRISPR tiling screen data Open
Tiling screens using CRISPR-Cas technologies provide a powerful approach to map regulatory elements to phenotypes of interest, but computational methods that effectively model these experimental approaches for different CRISPR technologies…
Multiparametric Whole Blood Dissection: A one‐shot comprehensive picture of the human hematopoietic system Open
Human hematopoiesis is a complex and dynamic system where morphologically and functionally diverse mature cell types are generated and maintained throughout life by bone marrow (BM) Hematopoietic Stem/Progenitor Cells (HSPC). Congenital an…
In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases Open
Hematopoietic stem/progenitor cells (HSPCs) are capable of supporting the lifelong production of blood cells exerting a wide spectrum of functions. Lentiviral vector HSPC gene therapy generates a human hematopoietic system stably marked at…
531. Computational Pipeline for the Identification of Integration Sites and Novel Method for the Quantification of Clone Sizes in Clonal Tracking Studies Open
Gene-corrected cells in Gene Therapy (GT) treated patients can be tracked in vivo by means of vector integration site (IS) analysis, since each engineered clone becomes univocally and stably marked by an individual IS. As the proper IS ide…