List of works - Francesca Ferrua

A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation

scientific article published on 21 April 2016

Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan.

scientific article published on 31 August 2009

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

scientific article published on 11 January 2017

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

scientific article published on 5 February 2014

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

scientific article published on 16 March 2015

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

scientific article published on 2 February 2018

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

scientific article published on 4 January 2018

Gene therapy for Wiskott-Aldrich Syndrome.

scientific article

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

scientific article

In vivo T-cell dynamics during immune reconstitution after hematopoietic stem cell gene therapy in adenosine deaminase severe combined immune deficiency ⬇️

scientific article published on April 7, 2011

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

scientific article published on 25 May 2016

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

scientific article

Pioglitazone as a novel therapeutic approach in chronic granulomatous disease.

scientific article published on 04 April 2016

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

scientific article published on 29 April 2011

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

scientific article published on 11 May 2010

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

scientific article published on 28 August 2017

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

scientific article

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

scientific article published on 29 April 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

scientific article

List of works by Francesca Ferrua

A novel genomic inversion in Wiskott-Aldrich-associated autoinflammation

Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan.

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

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

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

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

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

Gene therapy for Wiskott-Aldrich Syndrome.

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

In vivo T-cell dynamics during immune reconstitution after hematopoietic stem cell gene therapy in adenosine deaminase severe combined immune deficiency ⬇️

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

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

Pioglitazone as a novel therapeutic approach in chronic granulomatous disease.

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

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

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

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

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

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