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Lista de obras de Alessandro Aiuti

A combined immunodeficiency with severe infections, inflammation, and allergy caused by ARPC1B deficiency

artículo científico publicado en 2019

A highly efficacious lymphocyte chemoattractant, stromal cell-derived factor 1 (SDF-1)

artículo científico publicado en 1996

A map of human circular RNAs in clinically relevant tissues.

artículo científico publicado en 2017

A novel disorder involving dyshematopoiesis, inflammation, and HLH due to aberrant CDC42 function

artículo científico publicado en 2019

A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation

artículo científico publicado en 2016

A novel human packaging cell line with hematopoietic supportive capacity increases gene transfer into early hematopoietic progenitors.

artículo científico publicado en 2001

A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysis.

artículo científico publicado en 2016

ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency

artículo científico publicado en 2009

AQP8 transports NOX2-generated H2O2 across the plasma membrane to promote signaling in B cells.

artículo científico publicado en 2016

Abnormalities of acid-base balance and predisposition to metabolic acidosis in Metachromatic Leukodystrophy patients

artículo científico publicado en 2015

Advances in gene therapy for ADA-deficient SCID.

artículo científico publicado en 2002

Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID

artículo científico publicado en 2011

Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients

artículo científico publicado en 2017

Auto-antibodies against type I IFNs in patients with life-threatening COVID-19

artículo científico publicado en 2020

Autoimmune dysregulation and purine metabolism in adenosine deaminase deficiency

artículo científico publicado en 2012

Autoimmunity and regulatory T cells in 22q11.2 deletion syndrome patients.

artículo científico publicado en 2015

Autonomous role of Wiskott-Aldrich Syndrome platelet deficiency in inducing autoimmunity and inflammation

artículo científico publicado en 2018

B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients.

artículo científico publicado en 2014

B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome.

artículo científico publicado en 2015

Biased T-cell receptor repertoires in patients with chromosome 22q11.2 deletion syndrome (DiGeorge syndrome/velocardiofacial syndrome).

artículo científico publicado en 2003

Biological and functional characterization of bone marrow-derived mesenchymal stromal cells from patients affected by primary immunodeficiency.

artículo científico publicado en 2017

Bone marrow-derived CD34(-) fraction: A rich source of mesenchymal stromal cells for clinical application

artículo científico publicado en 2016

Burkitt's lymphoma in a patient with adenosine deaminase deficiency-severe combined immunodeficiency treated with polyethylene glycol-adenosine deaminase

artículo científico publicado en 2007

Cell-Surface Marking of CD34+-Restricted Phenotypes of Human Hematopoietic Progenitor Cells by Retrovirus-Mediated Gene Transfer ⬇️

artículo científico publicado el 1 de septiembre de 1997

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy

artículo científico publicado en 2012

Chronic granulomatous disease presenting with salmonella brain abscesses.

artículo científico publicado en 2014

Clinical applications of gene therapy for primary immunodeficiencies

artículo científico publicado en 2015

Clinical features and follow-up in patients with 22q11.2 deletion syndrome

artículo científico publicado en 2014

Combined immunodeficiency due to JAK3 mutation in a child presenting with skin granuloma.

artículo científico publicado en 2015

Complement activation is variably affected by fibronectin preparations obtained through different procedures

artículo científico publicado en 1988

Comprehensive genomic access to vector integration in clinical gene therapy

artículo científico publicado en 2009

Control of human coagulation by recombinant serine proteases. Blood clotting is activated by recombinant factor XII deleted of five regulatory domains.

artículo científico publicado en 1992

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

artículo científico publicado en 2002

Current understanding of the Wiskott-Aldrich syndrome and prospects for gene therapy.

artículo científico publicado en 2007

Decreased apoptosis of bone marrow progenitor cells in HIV-1-infected patients during highly active antiretroviral therapy

artículo científico publicado en 2004

Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapy

artículo científico publicado en 2012

Defective B-cell proliferation and maintenance of long-term memory in patients with chronic granulomatous disease.

artículo científico publicado en 2014

Defective Th1 cytokine gene transcription in CD4+ and CD8+ T cells from Wiskott-Aldrich syndrome patients.

artículo científico publicado en 2006

Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome

artículo científico publicado en 2011

Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry. ⬇️

artículo científico publicado en 2018

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.

artículo científico publicado en 2014

Early-onset monocyte–B–natural killer–dendritic cells’ deficiency successfully treated with hematopoietic stem cell transplantation

artículo científico publicado en 2011

Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation.

artículo científico publicado en 2006

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy

artículo científico publicado en 2017

Efficient gene transfer into primitive hematopoietic progenitors using a bone marrow microenvironment cell line engineered to produce retroviral vectors.

artículo científico publicado en 2004

Etiology, clinical outcome, and laboratory features in children with neutropenia: analysis of 104 cases

artículo científico publicado en 2013

Evaluation of ADA gene expression and transduction efficiency in ADA/SCID patients undergoing gene therapy.

artículo científico publicado en 2004

Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models

artículo científico publicado en 2009

Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects

artículo científico publicado en 2006

First Occurrence of Plasmablastic Lymphoma in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Disease Patient and Review of the Literature.

artículo científico publicado en 2018

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety

artículo científico publicado en 2018

Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

artículo científico publicado en 2017

Gene therapy for Wiskott-Aldrich Syndrome.

artículo científico publicado en 2014

Gene therapy for adenosine deaminase deficiency

artículo científico publicado en 2003

Gene therapy for adenosine deaminase deficiency

artículo científico publicado en 2010

Gene therapy for adenosine-deaminase-deficient severe combined immunodeficiency.

artículo científico publicado en 2004

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

artículo científico publicado en 2009

Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.

artículo científico publicado en 2017

Gene therapy for primary immunodeficiencies: Part 1.

artículo científico

Gene therapy for primary immunodeficiencies: Part 2.

artículo científico publicado en 2012

Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID.

artículo científico publicado en 2018

Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice.

artículo científico publicado en 2017

HIV type 1 protease inhibitors enhance bone marrow progenitor cell activity in normal subjects and in HIV type 1-infected patients

artículo científico publicado en 2005

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID.

artículo científico publicado en 2009

Hematopoietic support and cytokine expression of murine-stable hepatocyte cell lines (MMH).

artículo científico publicado en 1998

Hot spots of retroviral integration in human CD34+ hematopoietic cells

artículo científico publicado en 2007

Human CD26 expression in transgenic mice affects murine T-cell populations and modifies their subset distribution

scientific article published on 01 September 2002

Human CD34(+) cells express CXCR4 and its ligand stromal cell-derived factor-1. Implications for infection by T-cell tropic human immunodeficiency virus.

artículo científico publicado en 1999

Human CD4 produced in lymphoid cells of transgenic mice binds HIV gp120 and modifies the subsets of mouse T-cell populations

artículo científico publicado en 1993

Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity

artículo científico publicado en 2013

Human genetic and immunological determinants of critical COVID-19 pneumonia

artículo científico publicado en 2022

IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential.

artículo científico publicado en 2004

Identification of distinct elements of the stromal microenvironment that control human hematopoietic stem/progenitor cell growth and differentiation

artículo científico publicado en 1998

Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

artículo científico publicado en 2002

Immunotherapy of acute leukemia by chimeric antigen receptor-modified lymphocytes using an improved Sleeping Beauty transposon platform

artículo científico publicado en 2016

Impaired X-CGD T cell compartment is gp91phox-NADPH oxidase independent

artículo científico publicado en 2018

In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells

artículo científico publicado en 2015

Induction of CD4+ T cell depletion in mice doubly transgenic for HIV gp120 and human CD4

article

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

artículo científico publicado en 2011

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

artículo científico publicado en 2009

Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

artículo científico publicado en 2011

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

artículo científico publicado en 2016

Lack of evidence for a superantigen in lymphocytes from HIV-discordant monozygotic twins

artículo científico publicado en 1994

Large Deletion of MAGT1 Gene in a Patient with Classic Kaposi Sarcoma, CD4 Lymphopenia, and EBV Infection.

artículo científico publicado en 2016

Lentiviral Vector Gene Therapy Protects XCGD Mice From Acute Staphylococcus aureus Pneumonia and Inflammatory Response.

artículo científico publicado en 2016

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.

artículo científico publicado en 2016

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

artículo científico publicado en 2013

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

artículo científico publicado en 2013

Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction.

artículo científico publicado en 2004

Lentiviral vectors for the treatment of primary immunodeficiencies

artículo científico publicado en 2014

Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.

artículo científico publicado en 2006

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome

artículo científico publicado en 2011

Lentiviral-mediated gene therapy restores B cell tolerance in Wiskott-Aldrich syndrome patients

artículo científico publicado en 2015

Long-Term Outcome of Adenosine Deaminase-Deficient Patients-a Single-Center Experience.

artículo científico publicado en 2017

Long-term follow up of IPEX syndrome patients after different therapeutic strategies: an international multicenter retrospective study.

artículo científico publicado en 2017

Longitudinal Evaluation of Immune Reconstitution and B-cell Function After Hematopoietic Cell Transplantation for Primary Immunodeficiency.

artículo científico publicado en 2015

Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).

artículo científico publicado en 2007

Membrane expression of HLA-Cw4 free chains in activated T cells of transgenic mice

artículo científico publicado en 1995

Mobilized blood CD34+ cells transduced and selected with a clinically applicable protocol reconstitute lymphopoiesis in SCID-Hu mice.

artículo científico publicado en 2004

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

artículo científico publicado en 2007

Multiparametric Whole Blood Dissection: A one-shot comprehensive picture of the human hematopoietic system.

artículo científico publicado en 2017

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

artículo científico publicado en 2017

Neutrophils drive type-I interferon production and autoantibodies in Wiskott-Aldrich syndrome

artículo científico publicado en 2018

New perspectives in gene therapy for inherited disorders

scientific article published on 01 February 2020

Next-Generation Sequencing Reveals A JAGN1 Mutation in a Syndromic Child With Intermittent Neutropenia

scientific article published on 01 May 2019

Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.

artículo científico publicado en 2012

Pioglitazone as a novel therapeutic approach in chronic granulomatous disease.

artículo científico publicado en 2016

Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome

artículo científico publicado en 2012

Primary immunodeficiency diseases: Genomic approaches delineate heterogeneous Mendelian disorders

artículo científico publicado en 2016

Progress in gene therapy for primary immunodeficiencies using lentiviral vectors.

artículo científico

Purine metabolism, immune reconstitution, and abdominal adipose tumor after gene therapy for adenosine deaminase deficiency.

artículo científico publicado en 2011

Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

artículo científico publicado en 2009

Relevance of an academic GMP Pan-European vector infra-structure (PEVI).

artículo científico

Retroviral integrations in gene therapy trials

artículo científico publicado en 2012

Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: analysis of function and distribution in lymphoid organs.

artículo científico publicado en 2010

Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in ADA-SCID.

artículo científico publicado en 2010

SAP controls the cytolytic activity of CD8+ T cells against EBV-infected cells.

artículo científico publicado en 2005

Safer conditioning for blood stem cell transplants.

artículo científico publicado en 2016

Safety of retroviral gene marking with a truncated NGF receptor

scientific article published on 01 April 2003

Serratia marcescens Osteomyelitis in a Newborn with Chronic Granulomatous Disease

scientific article published on 01 August 2013

Severe Toxoplasma gondii infection in a member of a NFKB2-deficient family with T and B cell dysfunction.

artículo científico publicado en 2017

Skewed T-cell receptor repertoire, decreased thymic output, and predominance of terminally differentiated T cells in ataxia telangiectasia.

artículo científico publicado en 2002

Successful Treatment With Percutaneous Transhepatic Alcoholization of a Liver Abscess in a Child With Chronic Granulomatous Disease

artículo científico publicado en 2011

T-cell defects in patients with germline mutations account for combined immunodeficiency

artículo científico publicado en 2018

T-cell suicide gene therapy prompts thymic renewal in adults after hematopoietic stem cell transplantation.

artículo científico publicado en 2012

Targeted NGS Platforms for Genetic Screening and Gene Discovery in Primary Immunodeficiencies

article

Ten years of gene therapy for primary immune deficiencies.

artículo científico publicado en 2009

The 5' sequence of human factor XII gene contains transcription regulatory elements typical of liver specific, estrogen-modulated genes.

artículo científico publicado en 1993

The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function.

artículo científico publicado en 2009

The case of an APDS patient: Defects in maturation and function and decreased in vitro anti-mycobacterial activity in the myeloid compartment

artículo científico publicado en 2015

The chemokine SDF-1 is a chemoattractant for human CD34+ hematopoietic progenitor cells and provides a new mechanism to explain the mobilization of CD34+ progenitors to peripheral blood.

artículo científico publicado en 1997

The risk of COVID-19 death is much greater and age dependent with type I IFN autoantibodies

artículo científico publicado en 2022

The role of conditioning in hematopoietic stem cell gene therapy.

artículo científico publicado en 2016

Tracking genetically engineered lymphocytes long-term reveals the dynamics of T cell immunological memory.

artículo científico publicado en 2015

Transcriptional targeting of retroviral vectors to the erythroblastic progeny of transduced hematopoietic stem cells.

artículo científico publicado en 1999

Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug.

artículo científico publicado en 2017

Unpredictability of intravenous busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplantation for advanced beta thalassemia: limited toxicity with a dose-adjustment policy.

artículo científico publicado en 2009

Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.

artículo científico publicado en 2010

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

artículo científico publicado en 2016

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

artículo científico publicado en 2018

Wake-up Sleepy Gene: Reactivating Fetal Globin for β-Hemoglobinopathies

artículo científico publicado en 2018

Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans

artículo científico publicado en 2013

Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation.

artículo científico publicado en 2002

Wiskott-Aldrich syndrome protein-mediated actin dynamics control type-I interferon production in plasmacytoid dendritic cells

artículo científico publicado en 2013

[Gene therapy in pediatrics]

scientific article published on 01 December 2009

[Pediatric testing and primary immunodeficiencies]

scientific article published on 01 December 2009

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