A5010685005- Virus-based gene therapy research
- CRISPR and Genetic Engineering
- Hemoglobinopathies and Related Disorders
- RNA Interference and Gene Delivery
- CAR-T cell therapy research
- Muscle Physiology and Disorders
- Animal Genetics and Reproduction
- Pluripotent Stem Cells Research
- Developmental Biology and Gene Regulation
- Hemoglobin structure and function
- HIV Research and Treatment
- Prenatal Screening and Diagnostics
- Erythrocyte Function and Pathophysiology
- Skin and Cellular Biology Research
- Iron Metabolism and Disorders
- Viral Infectious Diseases and Gene Expression in Insects
- Hematopoietic Stem Cell Transplantation
- Ubiquitin and proteasome pathways
- Cytomegalovirus and herpesvirus research
- Immune Cell Function and Interaction
- Mesenchymal stem cell research
- Epigenetics and DNA Methylation
- Tissue Engineering and Regenerative Medicine
- Corneal Surgery and Treatments
- RNA modifications and cancer
University of Modena and Reggio Emilia
2015-2025
University of Padua
2023
Vital Therapies (United States)
2023
Genethon (France)
2012-2019
Institut des Maladies Génétiques Imagine
2018
Délégation Paris 5
2018
Université Paris Cité
2018
The Ohio State University Wexner Medical Center
2018
Sorbonne Paris Cité
2018
Inserm
2016-2017
Growth and repair of skeletal muscle are normally mediated by the satellite cells that surround fibers. In regenerating muscle, however, number myogenic precursors exceeds resident cells, implying migration or recruitment undifferentiated progenitors from other sources. Transplantation genetically marked bone marrow into immunodeficient mice revealed marrow-derived migrate areas induced degeneration, undergo differentiation, participate in regeneration damaged Genetically modified, could...
In allogeneic bone marrow transplantation (allo-BMT), donor lymphocytes play a central therapeutic role in both graft-versus-leukemia (GvL) and immune reconstitution. However, their use is limited by the risk of severe graft-versus-host disease (GvHD). Eight patients who relapsed or developed Epstein-Barr virus–induced lymphoma after T cell–depleted BMT were then treated with transduced herpes simplex virus thymidine kinase (HSV-TK) suicide gene. The survived for up to 12 months, resulting...
Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the first genetic disorder treated by gene therapy. Two different retroviral vectors were used to transfer ex vivo human ADA minigene into bone marrow cells and peripheral blood lymphocytes from two patients undergoing exogenous enzyme replacement After 2 years of treatment, long-term survival T B lymphocytes, cells, granulocytes expressing transferred was demonstrated resulted normalization immune repertoire...
It has recently been shown that mononuclear cells from murine skeletal muscle contain the potential to repopulate all major peripheral blood lineages in lethally irradiated mice, but origin of this activity is unknown. We have fractionated on basis hematopoietic markers show active population exclusively expresses stem cell antigens Sca-1 and CD45. Muscle obtained 6- 8-week-old C57BL/6-CD45.1 mice enriched for expressing CD45 were able generate not myogenic colonies vitro repopulated...
Human granulocyte colony stimulating factor (G-CSF) can support the survival and short term proliferation of interleukin 3 (IL 3)-dependent diploid murine hemopoietic progenitor cell line 32D C13. After 8 days in presence 30 U/ml G-CSF absence IL 3, great majority C13 cells becomes positive for myeloperoxidase (a marker that appears at promyelocytic stage granulocytic lineage) progressively differentiates into lactoferrin-containing neutrophilic granulocytes. Myeloperoxidase mRNA rapidly...
<h3>Importance</h3> Wiskott-Aldrich syndrome is a rare primary immunodeficiency associated with severe microthrombocytopenia. Partially HLA antigen–matched allogeneic hematopoietic stem cell (HSC) transplantation often curative but significant comorbidity. <h3>Objective</h3> To assess the outcomes and safety of autologous HSC gene therapy in syndrome. <h3>Design, Setting, Participants</h3> Gene-corrected HSCs were infused 7 consecutive patients lacking related or unrelated donors (age range,...
Abstract Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors attractive strategy to treat DMD. Here we show that locoregional systemic delivery of a rAAV2/8 vector expressing canine (cMD1) effective restoring expression stabilizing clinical symptoms studies performed on total 12 treated golden retriever...
BackgroundX-linked myotubular myopathy is a rare, life-threatening, congenital muscle disease observed mostly in males, which caused by mutations MTM1. No therapies are approved for this disease. We aimed to assess the safety and efficacy of resamirigene bilparvovec, an adeno-associated viral vector serotype 8 delivering human MTM1.MethodsASPIRO open-label, dose-escalation trial at seven academic medical centres Canada, France, Germany, USA. included boys younger than 5 years with X-linked...
We have investigated the effect of granulocyte colony-stimulating factor (G-CSF) delivery at site tumor growth by transducing, via retroviral vector, human (hu) G-CSF gene into colon adenocarcinoma C-26 and assaying ability transduced cells to form tumors when injected syngeneic mice. As a control, same were infected with retroviruses engineered transduce an unrelated gene, nerve receptor, or carry neomycin resistance only. Only huG-CSF unable develop tumors, although was expressed produced...
Human embryonic development involves transition from yolk sac (YS) to liver (L) hemopoiesis. We report the identification of pluripotent, erythroid, and granulo-macrophage progenitors in YS, L, blood human embryos. Furthermore, comprehensive studies are presented on number hemopoietic precursors, as well other cell types, at precisely sequential stages embryos early fetuses (i.e., 4.5-8 wk 9-10 postconception, respectively). Our results provide circumstantial support a monoclonal hypothesis...
Gene transfer into HSCs is an effective treatment for SCID, although potentially limited by the risk of insertional mutagenesis. We performed a genome-wide analysis retroviral vector integrations in genetically corrected and their multilineage progeny before up to 47 months after transplantation 5 patients with adenosine deaminase–deficient SCID. Gene-dense regions, promoters, transcriptionally active genes were preferred sites (RISs) both preinfusion transduced CD34+ cells vivo gene...
Nuclear localization of the Extradenticle (EXD) and PBX1 proteins is regionally restricted during Drosophila mammalian development. We studied subcellular EXD, PBX, their partners Homothorax (HTH) PREP1, in different cell contexts. HTH PREP1 are cytoplasmic require association with EXD/PBX for nuclear localization. EXD murine fibroblasts but not Schneider cells, which they actively exported to cytoplasm. Coexpression HTH/PREP1 causes heterodimers both propose that heterodimerization HTH/PREP...
Human limbal stem cells produce transit amplifying progenitors that migrate centripetally to regenerate the corneal epithelium. Coexpression of CCAAT enhancer binding protein delta (C/EBPdelta), Bmi1, and DeltaNp63alpha identifies mitotically quiescent cells, which generate holoclones in culture. Upon injury, a fraction these switches off C/EBPdelta proliferates, differentiates into mature cells. Forced expression inhibits growth colonies increases cell cycle length primary through activity...