G. Santilli

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Aliases
G Santilli, Giorgia Santilli
Publication record
92 papers · 3,242 citations · H-index 28

PAPERS

The future of ex vivo hematopoietic stem cell gene editing: what’s next
20251 citationsauthor #2
Unveiling the cut-and-repair cycle of designer nucleases in human stem and T cells via CLEAR-time dPCR
20251 citationsauthor #18
A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3
20250 citationsauthor #22
Tailoring capsid directed evolution technology for improved AAV-mediated CAR-T generation.
20243 citationsauthor #14
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia
20245 citationsauthor #19
Myelodysplasia and transgene inactivation in X-CGD-γ retroviral gene therapy: The usual suspects and new players.
20230 citationsauthor #2
Genomic investigations of unexplained acute hepatitis in children
202398 citationsauthor #47
Multidimensional Response Surface Methodology for the development of a gene editing protocol for p67phox-deficient Chronic Granulomatous Disease
20234 citationsauthor #10
An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells
202310 citationsauthor #6
AAV capsid bioengineering in primary human retina models
202312 citationsauthor #13
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression
202219 citationsauthor #11
EROS is a selective chaperone regulating the phagocyte NADPH oxidase and purinergic signalling
20228 citationsauthor #31
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors
202214 citationsauthor #24
Optimized cell culture conditions promote ex-vivo manipulation and expansion of primitive hematopoietic stem cells for therapeutic gene editing
20221 citationsauthor #6
Gene Editing in Human Haematopoietic Stem Cells for the Treatment of Primary Immunodeficiencies
20222 citationsauthor #3
GENOMIC INVESTIGATIONS OF ACUTE HEPATITIS OF UNKNOWN AETIOLOGY IN CHILDREN
202228 citationsauthor #29
Immunodeficiency, autoimmunity, and increased risk of B cell malignancy in humans with TRAF3 mutations
202228 citationsauthor #13
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease
20212 citationsauthor #18
EROS-mediated control of NOX2 and P2X7 biosynthesis
20214 citationsauthor #22
Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2
202035 citationsauthor #15
High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction
202076 citationsauthor #14
Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
2020100 citationsauthor #12
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice
202013 citationsauthor #12
Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
202014 citationsauthor #16
Lentiviral gene therapy for X-linked chronic granulomatous disease
2020196 citationsauthor #6
WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation
20208 citationsauthor #4
Restoring the natural tropism of AAV2 vectors for human liver
202042 citationsauthor #12
Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application
201942 citationsauthor #5
Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells
201942 citationsauthor #4
Effective Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)
20190 citationsauthor #6
EROS/CYBC1 mutations: Decreased NADPH oxidase function and chronic granulomatous disease.
201969 citationsauthor #12
Differential transgene silencing of myeloid-specific promoters in the AAVS1 safe harbor locus of iPSC-derived myeloid cells
20190 citationsauthor #4
Targeted gene correction for the treatment of Severe Combined Immunodeficiency caused by mutations in the IL7R gene
20190 citationsauthor #3
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality
201827 citationsauthor #10
British Society for Gene and Cell Therapy Annual Conference Friday 27th April 2018 Regent's Conference Centre, London, UK www.bsgct.org
20180 citationsauthor #75
Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease
201831 citationsauthor #3
Gene Therapy with pCCLchimP47-phox Lentiviral Vector Achieves Long-term Reconstitution of NADPH Oxidase Activity in p47-phox Chronic Granulomatous Disease (CGD) Mouse Model
20180 citationsauthor #9
EROS mutations: decreased NADPH oxidase function and chronic granulomatous disease
20180 citationsauthor #13
Pulmonary metastatic colonisation and granulomas in NOX2‐deficient mice
201831 citationsauthor #6
Targeted stem cells expressing TRAIL as a therapy for lung Cancer TACTICAL: a phase I/II trial
20186 citationsauthor #9
EROS is required for phagocyte NADPH oxidase function in humans and its deficiency causes Chronic Granulomatous Disease
20181 citationsauthor #12
Preclinical Safety and Efficacy Evaluation of Lentivirally transduced Hematopoietic Stem Cells for the treatment of Leukocyte Adhesion Deficiency type I.
20180 citationsauthor #13
Non-Clinical Efficacy and Safety Studies on G1XCGD, a lentiviral vector for ex vivo gene therapy of Chronic Granulomatous Disease
20180 citationsauthor #3
Targeted Gene Correction for the Treatment of Wiskott-Aldrich Syndrome
20170 citationsauthor #5
Characterization of a core region in the A2UCOE that confers effective anti-silencing activity
201711 citationsauthor #2
Development of a pCCLChim Lentiviral Vector for Gene Therapy of Patients with Chronic Granulomatous Disease (CGD) due to p47-phox Deficiency
20170 citationsauthor #8
A New Chapter on Targeted Gene Insertion for X-CGD: Do Not Skip the Intro(n).
20174 citationsauthor #1
Lentiviral Gene Therapy for p47(phox) Deficient Chronic Granulomatous Disease
20170 citationsauthor #12
P256 Targeted stem cells expressing trail as a therapy for lung cancer tactical: a phase i/ii trial
20170 citationsauthor #8
Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I
201620 citationsauthor #7
Establishing the platform for clinical gene therapy of p47phox chronic granulomatous disease (CGD)
20161 citationsauthor #9
Hyperinflammation in patients with chronic granulomatous disease leads to impairment of hematopoietic stem cell functions.
201670 citationsauthor #6
Patching up hematopoietic stem cells
20153 citationsauthor #1
Preclinical studies for the gene therapy of leukocyte adhesion deficiency type I
20150 citationsauthor #5
Hyperinflammation in Chronic Granulomatous Disease leads to 1 impairment of hematopoietic stem cell functions 2 3
20150 citationsauthor #6
Construction of stable packaging cell lines for clinical lentiviral vector
20150 citationsauthor #6
Construction of stable packaging cell lines for clinical lentiviral vector production
2015108 citationsauthor #7
IMPROVING STABILITY OF MEASLES AND MUMPS VACCINES BY REPLACING HUMAN SERUM ALBUMIN WITH AMINO ACIDS MIXES
20140 citationsauthor #6
Development of new lentiviral vectors for the gene therapy of LAD-I
20130 citationsauthor #3
Polyphenol E Enhances the Antitumor Immune Response in Neuroblastoma by Inactivating Myeloid Suppressor Cells
201366 citationsauthor #1
A clinical-grade constitutive packaging cell line for the production of self-inactivating lentiviral vectors
20132 citationsauthor #7
In vivo gentoxicity profile of a chimeric promoter driven lentiviral vector for X-CGD in mice
20130 citationsauthor #5
Catechins and antitumor immunity
20136 citationsauthor #1
Itgb2 hypomorphic mice: a novel approach for ex vivo LAD gene therapy
20120 citationsauthor #5
Gene Therapy for Primary Immunodeficiencies
201226 citationsauthor #2
FUNCTIONALLY ACTIVE MYELOID DERIVED SUPPRESSOR CELLS (MDSCS) ARE FOUND WITHIN THE BLOOD OF PATIENTS WITH NEUROBLASTOMA
20120 citationsauthor #5
Correction of SCID-X1 Using an Enhancerless Vav Promoter
201114 citationsauthor #3
Generation of Functional Neutrophils from a Mouse Model of X-Linked Chronic Granulomatous Disorder Using Induced Pluripotent Stem Cells
201128 citationsauthor #2
Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells.
2011151 citationsauthor #1
Development of a clinical trial of gene therapy for X-CGD using the pCCLCHIMGp91WPRE4 lentiviral vector
20110 citationsauthor #1
Targeted Lentiviral Gene Therapy to Rescue the X-CGD Phenotype
20100 citationsauthor #1
Gene-Corrected Neutrophils from Induced Pluripotent Stem Cells Derived from an X-CGD Disease Model
20100 citationsauthor #2
Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells
200964 citationsauthor #5
Clusterin, a haploinsufficient tumor suppressor gene in neuroblastomas.
200997 citationsauthor #6
Evaluation of self-inactivating lentiviral vectors containing myeloid specific promoters in combination with the ubiquitously acting chromatin opening element (UCOE) for gene therapy of X-CGD
20080 citationsauthor #1
Isolation and functional assessment of common, polymorphic variants of the B-MYB proto-oncogene associated with a reduced cancer risk
20084 citationsauthor #5
Isolation and functional assessment of common, polymorphic variants of the B-MYB proto-oncogene associated with a reduced cancer risk
20087 citationsauthor #5
Isolation and functional assessment of common, polymorphic variants of the B-MYB proto-oncogene associated with a reduced cancer risk (vol 27,pg 2929,2008)
20080 citationsauthor #5
Gene therapy of inherited immunodeficiencies
200815 citationsauthor #1
Temperature-dependent Modification and Activation of B-MYB
200529 citationsauthor #1
Essential requirement of apolipoprotein J (clusterin) signaling for IkappaB expression and regulation of NF-kappaB activity.
200366 citationsauthor #1
Essential requirement of Apolipoprotein J (clusterin) signalling for IKB expression and modulation of NF-kB activity: Implications for neuroblastoma
20030 citationsauthor #1
Transcription activation function of C/EBPalpha is required for induction of granulocytic differentiation.
2003109 citationsauthor #2
BCR/ABL activates mdm2 mRNA translation via the La antigen.
2003251 citationsauthor #7
Essential Requirement of Apolipoprotein J (Clusterin) Signaling for IκB Expression and Regulation of NF-κB Activity*
2003127 citationsauthor #1
Gene therapy of inherited immunodeficiencies.
200321 citationsauthor #1
hnRNP A1 Nucleocytoplasmic Shuttling Activity Is Required for Normal Myelopoiesis and BCR/ABL Leukemogenesis
2002126 citationsauthor #2
BCR-ABL suppresses C/EBPα expression through inhibitory action of hnRNP E2
2002295 citationsauthor #5
BCR-ABL suppresses C/EBPalpha expression through inhibitory action of hnRNP E2.
2002167 citationsauthor #5
PARP co-activates B-MYB through enhanced phosphorylation at cyclin/cdk2 sites
200128 citationsauthor #1
Urocortin Protects against Ischemic and Reperfusion Injury via a MAPK-dependent Pathway*
2000243 citationsauthor #4
Direct Transactivation of the Anti-apoptotic Gene Apolipoprotein J (Clusterin) by B-MYB*
2000114 citationsauthor #3

TIMELINE

2000 357 citations

2001 28 citations

2002 588 citations

2003 574 citations

2005 29 citations

2008 26 citations

2009 161 citations

2010 0 citations

2011 193 citations

2012 26 citations

2013 74 citations

2014 0 citations

2015 111 citations

2016 91 citations

2017 15 citations

2018 96 citations

2019 153 citations

2020 484 citations

2021 6 citations

2022 100 citations

2023 124 citations

2024 8 citations

2025 2 citations

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