Long-term lineage commitment in hematopoietic stem cell gene therapy

A Calabria, G Spinozzi, D Cesana, E Buscaroli… - Nature, 2024 - nature.com
Abstract Hematopoietic Stem Cell (HSC) gene therapy (GT) may provide lifelong
reconstitution of the hematopoietic system with gene-corrected cells1. However, the effects …

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

E Valeri, G Unali, F Piras, M Abou-Alezz, G Pais… - Molecular Therapy, 2024 - cell.com
Quiescent human hematopoietic stem cells (HSC) are ideal targets for gene therapy
applications due to their preserved stemness and repopulation capacities; however, they …

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

D Cesana, MP Cicalese, A Calabria, P Merli… - Nature …, 2024 - nature.com
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 …

Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome

G Consiglieri, F Tucci, M De Pellegrin… - Science Translational …, 2024 - science.org
Mucopolysaccharidosis type I Hurler (MPSIH) is characterized by severe and progressive
skeletal dysplasia that is not fully addressed by allogeneic hematopoietic stem cell …

Synergies at the Intersection: Exploring the Role of Bioinformatics in Enhancing Physiotherapy and Nursing Practice

M Abdulsalam, AA Hamisu, AM Ahmad, FB Wakili… - Biological …, 2024 - irrespub.com
In contemporary healthcare, integrating bioinformatics with physiotherapy and nursing
science has emerged as a promising approach to personalized patient care and clinical …

Modeling integration site data for safety assessment with MELISSA

TY Lin, G Ceoldo, K House, M Welty, TT Dang, D Klatt… - bioRxiv, 2024 - biorxiv.org
Gene and cell therapies pose safety concerns due to potential insertional mutagenesis by
viral vectors. We introduce MELISSA, a statistical framework for analyzing Integration Site …

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

I Merelli, S Beretta, D Cesana, A Gennari… - Database, 2023 - academic.oup.com
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 …

Spatio-temporal dynamics of the liver shapes hepatocytes heterogeneity and impacts in vivo gene transfer and editing

A Cantore, F Starinieri, M Milani, S Beretta, C Canepari… - 2023 - researchsquare.com
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 …