Nature Search

Longitudinal single-cell profiling of chemotherapy response in acute myeloid leukemia

Relapse within acute myeloid leukaemia may be driven by the presence of leukaemia stem cells. Here, the authors use single cell RNA-seq seq to characterise leukemia stem cells, and show miR-126 as a potential marker of resistance.

Matteo Maria Naldini
Gabriele Casirati
Bernhard Gentner
ResearchOpen Access08 Mar 2023
Nature Communications

Volume: 14, P: 1-20
Author Correction: Genotoxic effects of base and prime editing in human hematopoietic stem cells

Martina Fiumara
Samuele Ferrari
Luigi Naldini
Amendments and CorrectionsOpen Access25 Jan 2024
Nature Biotechnology

Volume: 42, P: 986
Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens

An immune suppressive tumor microenvironment (TME) is a limitation for immunotherapy. Here the authors show that, in a B cell acute lymphoblastic leukemia mouse model, gene-based delivery of IFNα reprograms the leukemia-induced immunosuppressive TME into immunostimulatory and enhances T-cell responses.

Giulia Escobar
Luigi Barbarossa
Luigi Naldini
ResearchOpen Access24 Jul 2018
Nature Communications

Volume: 9, P: 1-16
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
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

Brian D Brown
Bernhard Gentner
Luigi Naldini
Research16 Nov 2007
Nature Biotechnology

Volume: 25, P: 1457-1467
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID

Leukaemia development has been reported as an associated risk of haematopoietic stem cell gene therapy (HSPC-GT) using retroviral vectors in different diseases. Here, the authors show a case of T-cell acute lymphoid leukaemia in a patient with Adenosine Deaminase-deficient Severe Combined Immunodeficiency (ADA-SCID) treated with retroviral gene therapy.

Daniela Cesana
Maria Pia Cicalese
Alessandro Aiuti
ResearchOpen Access30 Apr 2024
Nature Communications

Volume: 15, P: 1-18
Stable knockdown of microRNA in vivo by lentiviral vectors

To study microRNA function in vivo, the authors optimize lentiviral-driven expression of microRNA target sequences in mice and show dose-dependent inhibition of microRNA-mediated regulation of reporter constructs as well as of natural microRNA targets. With the inhibition of a miR-223, they can phenocopy the knockout of this microRNA.

Bernhard Gentner
Giulia Schira
Luigi Naldini
Research30 Nov 2008
Nature Methods

Volume: 6, P: 63-66
miR-126 identifies a quiescent and chemo-resistant human B-ALL cell subset that correlates with minimal residual disease

Carolina Caserta
Silvia Nucera
Bernhard Gentner
Research28 Aug 2023
Leukemia

Volume: 37, P: 1994-2005
Genotoxic effects of base and prime editing in human hematopoietic stem cells

Base and prime editors induce double-strand breaks, deletions and translocations in hematopoietic cells.

Martina Fiumara
Samuele Ferrari
Luigi Naldini
ResearchOpen Access07 Sept 2023
Nature Biotechnology

Volume: 42, P: 877-891
CD14 positive cells accelerate hematopoietic stem cell engraftment

Alice Pievani
Valentina Granata
Marta Serafini
Research08 Apr 2022
Bone Marrow Transplantation

Volume: 57, P: 942-948
Targeted genome editing in human repopulating haematopoietic stem cells

The feasibility of targeted genome editing in human haematopoietic stem cells is demonstrated; the study overcomes previously existing barriers by tailoring delivery platforms and culture conditions.

Pietro Genovese
Giulia Schiroli
Luigi Naldini
Research28 May 2014
Nature

Volume: 510, P: 235-240
Cellular and transcriptional dynamics of human neutrophils at steady state and upon stress

Recent studies suggest that neutrophils can exhibit substantial function diversity. Here, Ostuni and colleagues perform immunophenotyping and transcriptome analysis to characterize the heterogeneity of human neutrophils, both under steady state and upon stress-induced conditions.

Elisa Montaldo
Eleonora Lusito
Renato Ostuni
Research22 Sept 2022
Nature Immunology

Volume: 23, P: 1470-1483
Immune signature drives leukemia escape and relapse after hematopoietic cell transplantation

Post-transplantation relapse in acute myeloid leukemia patients without genomic loss of HLA is driven by transcriptional alterations in antigen presentation and T cell costimulation genes.

Cristina Toffalori
Laura Zito
Luca Vago
Research25 Mar 2019
Nature Medicine

Volume: 25, P: 603-611
Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I

Isabella Azario
Alice Pievani
Marta Serafini
ResearchOpen Access25 Aug 2017
Scientific Reports

Volume: 7, P: 1-13
Allogeneic hematopoietic stem cell transplantation in patients older than 65 years with acute myeloid leukemia and myelodysplastic syndrome: a 15-year experience

Simona Piemontese
Lorenzo Lazzari
Fabio Ciceri
Correspondence05 Feb 2022
Bone Marrow Transplantation

Volume: 57, P: 678-680

Search

Longitudinal single-cell profiling of chemotherapy response in acute myeloid leukemia

Author Correction: Genotoxic effects of base and prime editing in human hematopoietic stem cells

Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens

Long-term lineage commitment in haematopoietic stem cell gene therapy

Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

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

Stable knockdown of microRNA in vivo by lentiviral vectors

miR-126 identifies a quiescent and chemo-resistant human B-ALL cell subset that correlates with minimal residual disease

Genotoxic effects of base and prime editing in human hematopoietic stem cells

CD14 positive cells accelerate hematopoietic stem cell engraftment

Targeted genome editing in human repopulating haematopoietic stem cells

Cellular and transcriptional dynamics of human neutrophils at steady state and upon stress

Immune signature drives leukemia escape and relapse after hematopoietic cell transplantation

Neonatal umbilical cord blood transplantation halts skeletal disease progression in the murine model of MPS-I

Allogeneic hematopoietic stem cell transplantation in patients older than 65 years with acute myeloid leukemia and myelodysplastic syndrome: a 15-year experience

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