Federico Mingozzi Selected Research

Factor IX (Coagulation Factor IX)

1/2022	Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice.
1/2020	Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
12/2019	Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice.
11/2014	Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
7/2012	Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.
3/2012	Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.
12/2011	Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
7/2010	Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.
6/2010	Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
6/2009	Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors.

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Federico Mingozzi Research Topics

Disease

19	Hemophilia B (Haemophilia B) 01/2022 - 01/2004
11	Glycogen Storage Disease Type II (Pompe's Disease) 11/2021 - 10/2012
9	Hemorrhage 01/2022 - 08/2004
7	Hemophilia A (Haemophilia) 11/2021 - 03/2006
6	Infections 07/2020 - 10/2007
4	Inflammation (Inflammations) 10/2021 - 01/2013
4	Inborn Genetic Diseases (Disease, Hereditary) 01/2018 - 05/2003
3	Protein Deficiency 10/2019 - 08/2004
3	Mucopolysaccharidosis III (Sanfilippo Syndrome) 04/2015 - 12/2012
2	Crigler-Najjar Syndrome 03/2021 - 01/2016
2	Hepatocellular Carcinoma (Hepatoma) 09/2020 - 01/2020
2	Neoplasms (Cancer) 07/2020 - 03/2011
2	Fabry Disease (Fabry's Disease) 01/2018 - 01/2015
2	Blindness (Hysterical Blindness) 08/2016 - 11/2009
2	Neuroinflammatory Diseases 04/2015 - 12/2012
2	Lysosomal Storage Diseases (Lysosomal Storage Disease) 04/2015 - 01/2015
2	Hypersensitivity (Allergy) 01/2013 - 01/2013
2	Autoimmune Diseases (Autoimmune Disease) 01/2013 - 01/2013
2	Activated Protein C Resistance (APC Resistance) 03/2005 - 06/2003
2	Venous Thromboembolism 03/2005 - 06/2003
1	Maple Syrup Urine Disease 01/2022
1	Inborn Errors Metabolism (Inborn Errors of Metabolism) 01/2022
1	Perinatal Death 01/2022
1	Familial Hypophosphatemic Rickets (X-Linked Hypophosphatemia) 10/2021
1	Congenital Abnormalities (Deformity) 10/2021
1	Down Syndrome (Down's Syndrome) 10/2021
1	Brain Diseases (Brain Disorder) 10/2021
1	Uveitis 10/2021
1	Coma (Comas) 03/2021
1	Hyperbilirubinemia 03/2021
1	Ornithine Carbamoyltransferase Deficiency Disease 03/2021
1	von Willebrand Diseases (von Willebrand's Disease) 01/2021
1	Thrombotic Microangiopathies 07/2020
1	Thrombosis (Thrombus) 07/2020
1	Parkinson Disease (Parkinson's Disease) 01/2020
1	Sarcoglycanopathies 12/2019
1	Vacuolar myopathy 10/2019
1	Muscle Weakness 01/2018
1	Neuromuscular Manifestations 01/2018
1	Glycogen Storage Disease Type III (Cori's Disease) 01/2018
1	Familial Dysautonomia (Riley Day Syndrome) 01/2018
1	Cardiomegaly (Heart Hypertrophy) 11/2017
1	Wasting Syndrome (Wasting Disease) 01/2017
1	Muscular Dystrophies (Muscular Dystrophy) 01/2017
1	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 01/2017
1	Congenital Structural Myopathies (Centronuclear Myopathy) 01/2017
1	Metabolic Diseases (Metabolic Disease) 01/2016

Drug/Important Bio-Agent (IBA)

17	Factor IX (Coagulation Factor IX)FDA LinkGeneric 01/2022 - 01/2004
10	AcidsIBA 11/2021 - 10/2012
10	EnzymesIBA 11/2021 - 01/2015
7	GlucosidasesIBA 11/2020 - 10/2012
6	AntibodiesIBA 01/2020 - 07/2012
6	AntigensIBA 01/2013 - 08/2004
5	Retinaldehyde (Retinal)IBA 08/2016 - 05/2008
4	alpha-Glucosidases (Acid Maltase)IBA 11/2021 - 03/2019
4	Proteins (Proteins, Gene)FDA Link 01/2020 - 11/2009
3	Factor VIII (Coagulation Factor VIII)IBA 11/2021 - 03/2005
3	GlycogenIBA 01/2020 - 11/2017
3	DNA (Deoxyribonucleic Acid)IBA 01/2015 - 03/2011
2	von Willebrand FactorIBA 01/2021 - 07/2020
2	AnticoagulantsIBA 07/2020 - 01/2020
2	Blood Coagulation Factors (Coagulation Factor)IBA 01/2020 - 01/2016
2	Glucose (Dextrose)FDA LinkGeneric 01/2020 - 05/2013
2	EpitopesIBA 12/2019 - 10/2012
2	Pharmaceutical PreparationsIBA 01/2018 - 11/2016
2	human GAA protein (Myozyme)FDA Link 11/2016 - 10/2012
2	Lysergic Acid Diethylamide (LSD)IBA 04/2015 - 01/2013
2	GlycosaminoglycansIBA 04/2015 - 01/2013
2	N-sulfoglucosamine sulfohydrolase (sulfamidase)IBA 01/2013 - 12/2012
1	Branched-Chain Amino AcidsIBA 01/2022
1	Complementary DNA (cDNA)IBA 01/2022
1	Factor VII (Proconvertin)IBA 11/2021
1	Fibroblast Growth Factor-23IBA 10/2021
1	ChloridesIBA 10/2021
1	gamma-Aminobutyric Acid (GABA)IBA 10/2021
1	Immunologic Factors (Immunomodulators)IBA 10/2021
1	Uridine Diphosphate (UDP)IBA 03/2021
1	Glucuronosyltransferase (UDP Glucuronosyltransferase)IBA 03/2021
1	AmmoniaIBA 03/2021
1	Valoctocogene RoxaparvovecIBA 01/2021
1	BilirubinIBA 09/2020
1	Factor V (Coagulation Factor V)IBA 07/2020
1	Hemostatics (Antihemorrhagics)IBA 07/2020
1	Untranslated RNA (Noncoding RNA)IBA 01/2020
1	Single-Domain AntibodiesIBA 01/2020
1	Glial Cell Line-Derived Neurotrophic Factor (GDNF)IBA 01/2020
1	AntithrombinsIBA 01/2020
1	Transaminases (Aminotransferases)IBA 01/2020
1	Fetal Proteins (Fetoprotein)IBA 01/2020
1	Sarcoglycans (beta Sarcoglycan)IBA 12/2019
1	Glycogen Debranching Enzyme System (Glycogen Debranching Enzyme)IBA 01/2018
1	myotubularinIBA 01/2017
1	DesminIBA 01/2017
1	DystrophinIBA 01/2017
1	UGT1A1 enzymeIBA 01/2016

Therapy/Procedure

12	Therapeutics 01/2020 - 05/2003
6	Enzyme Replacement Therapy 11/2017 - 10/2012
1	Organ Transplantation 01/2022
1	Surgical Instruments (Clip) 01/2022
1	Intravitreal Injections 10/2021