Update README.md

README.md

-# Zonari_mPBHSCexp_2025_BulkRNAseq
+# BulkRNAseq analyses
+
+This repository contains the key scripts used to generate the final tables and figures featured in the accompanying manuscript:  
+**Zonari et al**, _Expansion of Hematopoietic Stem and Progenitor Cells from Human Mobilized Peripheral Blood for Gene Therapy_, XXX, 2025.  
+
+## Abstract 
+Ex vivo expansion of mobilized peripheral blood (mPB) hematopoietic stem cells (HSCs)
+represents a promising approach to advance cell and gene therapy strategies yet is hampered by
+loss of stem cell function when applying commonly used culture protocols. We performed an indepth
+characterization of mPB expansion cultures by single cell RNA sequencing, which
+highlighted differentiation trajectories with preservation of lineage fidelity in committed
+progenitors. Defining a putative HSC cluster allowed an estimation of transduction efficiency in
+ex vivo cultures, which correlated with long-term gene marking in xenografts and patients enrolled
+in a gene therapy study. We then developed a clinically translatable, GMP-compliant process to
+expand lentivirus (LV)-transduced HSCs from mPB of pediatric patients and adult donors, by
+biologically informed protocol improvements of cytokine supplementation, media choice, timing
+of LV transduction and combinations of small molecules preventing the activation of
+differentiation programs. Our optimized process outperforms validated state-of-the-art cord blood
+expansion protocols when applied to mPB. LV integration site analysis and genomic barcodebased
+clonal tracking provided definitive proof for symmetric HSC self-renewal divisions
+occurring during ex vivo culture. These results warrant clinical testing of this HSC
+transduction/expansion process in an upcoming clinical gene therapy trial for autosomal recessive
+osteopetrosis (EU CT 2024-518972-30), addressing the need of obtaining a high number of
+functioning osteoclast progenitors for rapid bone marrow niche remodeling, where gene-corrected
+HSC can engraft and allow long-term disease correction.
+
+GEO: [GSE292605](https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE292605). 
 
 Below a brief description with Bulk and scRNAseq workflow adopted in this work.