Nature Search

Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia

Here, the authors show how in β-thalassemia, a perturbed bone marrow microenvironment leads to altered hematopoiesis. Reduced TGFβ reduces autophagy levels, in turn reducing dormancy and priming of haematopoietic stem cells and multipotent progenitors towards erythroid lineage.

Maria Rosa Lidonnici
Giulia Chianella
Giuliana Ferrari
ResearchOpen Access01 Jul 2025
Nature Communications

Volume: 16, P: 1-19
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

In a phase 1/2 clinical trial, gene therapy with autologous hematopoietic stem cells significantly reduced transfusion requirement in adults and children with transfusion dependent ß-thalassemia.

Sarah Marktel
Samantha Scaramuzza
Giuliana Ferrari
Research21 Jan 2019
Nature Medicine

Volume: 25, P: 234-241
Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells

Fulvio Mavilio
Graziella Pellegrini
Michele De Luca
Research19 Nov 2006
Nature Medicine

Volume: 12, P: 1397-1402
Failure to correct murine muscular dystrophy

Giuliana Ferrari
Anna Stornaiuolo
Fulvio Mavilio
Research28 Jun 2001
Nature

Volume: 411, P: 1014-1015
Long-term lineage commitment in haematopoietic stem cell gene therapy

Haematopoietic stem cell (HSC) clonogenic activity, lineage output, lineage commitment and somatic mutation rates are influenced by the underlying disease, patient age, extent of genetic defect correction and hematopoietic stress imposed by the inherited disease, suggesting HSC adaptation.

Andrea Calabria
Giulio Spinozzi
Eugenio Montini
ResearchOpen Access23 Oct 2024
Nature

Volume: 636, P: 162-171
Transcript imaging of the development of human T helper cells using oligonucleotide arrays

Lars Rogge
Elisabetta Bianchi
Ulrich Certa
Research01 May 2000
Nature Genetics

Volume: 25, P: 96-101
Community voices: sowing, germinating, flourishing as strategies to support inclusion in STEM

Understanding gaps in academic representation while considering the intersectionality concept is paramount to promoting real progress towards a more inclusive STEM. Here we discuss ways in which STEM careers can be sown and germinated so that inclusivity can flourish.

Luisa Maria Diele-Viegas
Thamara Santos de Almeida
Flávia Virginio
Comments & OpinionOpen Access09 Jun 2022
Nature Communications

Volume: 13, P: 1-5
Gene therapy using haematopoietic stem and progenitor cells

Haematopoietic stem and progenitor cell (HSPC) gene therapy using lentiviral or gammaretroviral vectors has now been approved for clinical use. In this Review, Ferrari, Thrasher and Aiuti discuss the history of HSPC gene therapy, the clinical promise of gene-editing HPSCs and the use of HSPC gene therapy to treat specific diseases.

Giuliana Ferrari
Adrian J. Thrasher
Alessandro Aiuti
Reviews10 Dec 2020
Nature Reviews Genetics

Volume: 22, P: 216-234
Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

Alessandro Aiuti
Sergio Vai
Claudio Bordignon
Correspondence01 May 2002
Nature Medicine

Volume: 8, P: 423-425
Donor-derived CARCIK-CD19 cells engineered with Sleeping Beauty transposon in acute lymphoblastic leukemia relapsed after allogeneic transplantation

Federico Lussana
Chiara F. Magnani
Andrea Biondi
ResearchOpen Access03 Apr 2025
Blood Cancer Journal

Volume: 15, P: 1-14
Development of an Injectable Slow-Release Metformin Formulation and Evaluation of Its Potential Antitumor Effects

Sara Baldassari
Agnese Solari
Tullio Florio
ResearchOpen Access02 Mar 2018
Scientific Reports

Volume: 8, P: 1-12
Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment

Oriana Romano
Clelia Peano
Annarita Miccio
ResearchOpen Access20 Apr 2016
Scientific Reports

Volume: 6, P: 1-15
Phylogeography and genomic epidemiology of SARS-CoV-2 in Italy and Europe with newly characterized Italian genomes between February-June 2020

Alessia Lai
Annalisa Bergna
Dario Russignaga
ResearchOpen Access06 Apr 2022
Scientific Reports

Volume: 12, P: 1-12

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