Francesca Ferrua Selected Research

Severe combined immunodeficiency due to adenosine deaminase deficiency

1/2021	Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guérin infection.
1/2020	Urogenital Abnormalities in Adenosine Deaminase Deficiency.
12/2019	Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.
12/2018	Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis.
1/2018	Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.
11/2017	Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug.
11/2017	Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients.
1/2016	Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.
3/2014	B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients.
6/2011	Purine metabolism, immune reconstitution, and abdominal adipose tumor after gene therapy for adenosine deaminase deficiency.

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Francesca Ferrua Research Topics

Disease

12	Severe combined immunodeficiency due to adenosine deaminase deficiency 01/2021 - 01/2009
11	Severe Combined Immunodeficiency (Bare Lymphocyte Syndrome) 01/2022 - 01/2009
5	Wiskott-Aldrich Syndrome 01/2022 - 08/2013
4	Infections 01/2021 - 11/2009
2	Type 1 Hyper-IgM Immunodeficiency Syndrome 01/2021 - 01/2019
2	Metachromatic Leukodystrophy (Sulfatide Lipidosis) 12/2019 - 01/2018
2	Hemorrhage 05/2019 - 01/2019
1	Paralysis (Palsy) 02/2022
1	Immune System Diseases (Immune Disorders) 01/2022
1	Mucopolysaccharidosis I (Hurler Syndrome) 11/2021
1	Hemophagocytic Lymphohistiocytosis (Hemophagocytic Syndrome) 01/2021
1	Down Syndrome (Down's Syndrome) 01/2021
1	Tuberculosis (Tuberculoses) 01/2021
1	Cryptorchidism (Undescended Testes) 01/2020
1	COVID-19 01/2020
1	Metabolic Diseases (Metabolic Disease) 01/2016
1	Chronic Granulomatous Disease 01/2016
1	Neoplasms (Cancer) 06/2011
1	Iron Overload 11/2009
1	Hepatic Veno-Occlusive Disease (Disease, Hepatic Veno-Occlusive) 11/2009
1	beta-Thalassemia (Cooley's Anemia) 11/2009
1	Liver Cirrhosis (Hepatic Cirrhosis) 11/2009

Drug/Important Bio-Agent (IBA)

9	Adenosine DeaminaseIBA 01/2021 - 01/2009
3	purineIBA 11/2017 - 01/2009
2	AntigensIBA 01/2021 - 11/2009
2	pegademase bovine (pegADA)FDA Link 01/2020 - 11/2017
2	Proteins (Proteins, Gene)FDA Link 01/2019 - 08/2013
2	Busulfan (Busulfex)FDA Link 10/2010 - 11/2009
1	Messenger RNA (mRNA)IBA 02/2022
1	COVID-19 VaccinesIBA 02/2022
1	2019-nCoV Vaccine mRNA-1273IBA 02/2022
1	VaccinesIBA 02/2022
1	EmapalumabIBA 01/2021
1	Monoclonal AntibodiesIBA 01/2021
1	Biological ProductsIBA 01/2021
1	Immunoglobulin G (IgG)IBA 01/2021
1	Immunoglobulins (Immunoglobulin)IBA 01/2020
1	Wiskott-Aldrich Syndrome ProteinIBA 05/2019
1	CD40 Ligand (CD40L)IBA 01/2019
1	sofosbuvir drug combination ledipasvirIBA 12/2018
1	defibrotideIBA 01/2018
1	Pharmaceutical PreparationsIBA 11/2017
1	HLA Antigens (Human Leukocyte Antigens)IBA 11/2017
1	Adenosine (Adenocard)FDA LinkGeneric 11/2017
1	Pioglitazone (Actos)FDA Link 01/2016
1	fludarabineIBA 10/2010

Therapy/Procedure

5	Hematopoietic Stem Cell Transplantation 01/2022 - 01/2019
2	Aftercare (After-Treatment) 01/2020 - 11/2017
2	Stem Cell Transplantation 11/2017 - 11/2009
1	Cell Transplantation 01/2022
1	Therapeutics 01/2021
1	Orchiopexy 01/2020
1	Platelet Transfusion (Blood Platelet Transfusions) 05/2019
1	Enzyme Replacement Therapy 11/2017
1	Bone Marrow Transplantation (Transplantation, Bone Marrow) 10/2010