Lista de obras - Fulvio Mavilio - Dominio Público Uruguay

A human homoeo box gene specifically expressed in spinal cord during embryonic development

artículo científico publicado en 1986

A new abnormal human hemoglobin: Hb Prato (alpha 2 31 (B12) Arg leads to Ser beta 2)

artículo científico publicado en 1979

A new human hemoglobin variant: Hb BARI (alpha 2 45 (CD3) His leads to Gln beta 2)

artículo científico publicado en 1980

A single epidermal stem cell strategy for safe ex vivo gene therapy.

artículo científico publicado en 2015

Absence of an intrathecal immune reaction to a helper-dependent adenoviral vector delivered into the cerebrospinal fluid of non-human primates.

artículo científico publicado en 2007

Alternative splicing caused by lentiviral integration in the human genome

artículo científico publicado en 2012

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

artículo científico publicado en 2018

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

scientific article published on 13 September 2019

C/EBPdelta regulates cell cycle and self-renewal of human limbal stem cells.

artículo científico publicado en 2007

Characterization of Two Neuroblastoma Cell Lines Expressing Recombinant Nerve Growth Factor Receptors ⬇️

artículo científico publicado el 1 de enero de 1991

Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor

artículo científico publicado en 2005

Comprehensive genomic access to vector integration in clinical gene therapy

artículo científico publicado en 2009

Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.

artículo científico publicado en 2010

Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.

artículo científico publicado en 2006

Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted lentiviral vectors

artículo científico publicado en 2008

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning

artículo científico publicado en 2011

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.

artículo científico publicado en 2017

Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors

artículo científico publicado en 2013

Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry.

artículo científico publicado en 2017

Differential and stage-related expression in embryonic tissues of a new human homoeobox gene

artículo científico publicado en 1986

Differential regulation by retinoic acid of the homeobox genes of the four HOX loci in human embryonal carcinoma cells ⬇️

artículo científico publicado el 1 de marzo de 1991

Differential regulation of transferrin receptor gene expression in human hemopoietic cells: molecular and cellular aspects

scientific article published on 01 January 1987

Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation.

artículo científico publicado en 2016

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

artículo científico publicado en 2020

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

artículo científico publicado en 2016

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.

artículo científico publicado en 2018

Epithelial stem cells in corneal regeneration and epidermal gene therapy

scientific article published on January 2009

Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors

artículo científico

Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in nonhuman primates.

artículo científico publicado en 2017

Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair

artículo científico publicado en 2018

Failure to correct murine muscular dystrophy

artículo científico publicado en 2001

Functional dissection of a transcriptionally active, target-specific Hox-Pbx complex

artículo científico publicado en 1997

Gene Therapy Approaches to Hemoglobinopathies.

artículo científico publicado en 2017

Gene Therapy for Hemoglobinopathies

article

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study

scientific article published on 01 October 2018

Gene therapy approaches for epidermolysis bullosa

artículo científico publicado en 2005

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

artículo científico publicado en 2017

Gene therapy in combination with tissue engineering to treat epidermolysis bullosa.

artículo científico publicado en 2006

Gene therapy of inherited skin adhesion disorders

article

Gene therapy of inherited skin adhesion disorders: a critical overview.

artículo científico publicado en 2009

Gene therapy of skin adhesion disorders (mini review).

artículo científico publicado en 2012

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

artículo científico publicado en 2014

Gene therapy: back on track?

artículo científico publicado en 2010

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency

artículo científico publicado en 2017

Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.

artículo científico publicado en 2015

Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells.

artículo científico publicado en 2014

Genomic analysis of Sleeping Beauty transposon integration in human somatic cells

artículo científico publicado en 2014

Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination

artículo científico publicado en 2013

HOX gene activation by retinoic acid ⬇️

artículo científico publicado el 1 de octubre de 1991

HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia

artículo científico publicado en 1997

Haemoglobin Lepore Trait: Haematological and Structural Studies on the Italian Population ⬇️

artículo científico publicado el 1 de agosto de 1979

Haemoglobin switching in human embryos: asynchrony of zeta----alpha and epsilon----gamma-globin switches in primitive and definite erythropoietic lineage

artículo científico publicado en 1985

Hb J Baltimore (beta 16 (A13) Gly leads to Asp) in association with beta-thalassemia in a Sicilian family

artículo científico publicado en 1979

Hemoglobin Gavello - alpha 2 beta 2 47 (CD6) Asp replaced by Gly. A new hemoglobin variant from Polesine (Italy)

artículo científico publicado en 1977

Hemoglobin Legnano (alpha2 141 (HC3) Arg replaced by Leu beta2): a new abnormal human hemoglobin with high oxygen affinity

artículo científico publicado en 1978

Hemoglobin bologna (alpha 2 beta 2 61 (E5) lys replaced by met). An abnormal human hemoglobin with low oxygen affinity

scientific article published on 01 April 1981

Heterocellular hereditary persistence of fetal hemoglobin (HPFH). Molecular mechanisms of abnormal ?-gene expression in association with ? thalassemia and linkage relationship with the ?-globin gene cluster

article

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion

artículo científico publicado en 2010

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.

artículo científico publicado en 2010

High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors

artículo científico publicado en 2001

Hot spots of retroviral integration in human CD34+ hematopoietic cells

artículo científico publicado en 2007

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.

artículo científico publicado en 2012

IL4 gene delivery to the CNS recruits regulatory T cells and induces clinical recovery in mouse models of multiple sclerosis.

artículo científico publicado en 2008

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

artículo científico publicado en 2018

Inhibition of retinoic acid-induced activation of 3' human HOXB genes by antisense oligonucleotides affects sequential activation of genes located upstream in the four HOX clusters.

artículo científico publicado en 1994

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

artículo científico publicado en 2011

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

artículo científico publicado en 2009

Interactions between Retroviruses and the Host Cell Genome.

artículo científico publicado en 2017

Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts

artículo científico publicado en 2012

Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy

artículo científico publicado en 2007

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

artículo científico publicado en 2017

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

artículo científico publicado en 2007

Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

article

Muscle regeneration by bone marrow-derived myogenic progenitors

artículo científico publicado en 1998

Muscle-derived hematopoietic stem cells are hematopoietic in origin

artículo científico publicado en 2002

Nuclear architecture dictates HIV-1 integration site selection.

artículo científico publicado en 2015

Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication.

artículo científico publicado en 2013

Occurrence of Hb J Paris in an Italian family and recombination studies on the free abnormal alpha-chain

artículo científico publicado en 1979

Occurrence of Hb M Iwate (alpha 2 87 His leads to Tyr beta 2) in an Italian carrier

artículo científico publicado en 1981

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

scientific article published on 17 October 2018

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome

artículo científico publicado en 2015

PPARdelta is a ligand-dependent negative regulator of vitamin D3-induced monocyte differentiation

artículo científico publicado en 2008

Perspectives on best practices for gene therapy programs

artículo científico publicado en 2015

Post-translational control of human hemoglobin synthesis: the role of the differential affinity between globin chains in the control of mutated globin gene expression

artículo científico publicado en 1980

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

artículo científico publicado en 2018

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

artículo científico publicado en 2018

Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells.

artículo científico publicado en 2011

Prep1, a novel functional partner of Pbx proteins.

artículo científico publicado en 1998

Regulation of vertebrate homeobox-containing genes by morphogens.

artículo científico publicado en 1993

Repairing without cutting: a safer alternative to gene correction?

artículo científico publicado en 2014

Retinoic acid induces stage-specific antero-posterior transformation of rostral central nervous system.

artículo científico publicado en 1995

Retroviral Scanning: Mapping MLV Integration Sites to Define Cell-specific Regulatory Regions

artículo científico publicado en 2017

Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells.

artículo científico publicado en 2006

Reversible immortalization of human myogenic cells by site-specific excision of a retrovirally transferred oncogene.

artículo científico publicado en 1999

Risk assessment in skin gene therapy: viral-cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors

artículo científico publicado en 2011

Role of CD34 antigen in myeloid differentiation of human hematopoietic progenitor cells

artículo científico publicado en 2008

Safety of retroviral gene marking with a truncated NGF receptor

scientific article published on 01 April 2003

Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes.

artículo científico publicado en 2013

Site-specific integration into the human genome: ready for clinical application?

artículo científico publicado en 2006

Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectors.

artículo científico publicado en 2004

Studies on a family with Hb J Calabria (alpha 2 beta 2 64 (E8) Gly replaced by Asp)

artículo científico publicado en 1979

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

artículo científico publicado en 2017

T Lymphocytes transduced with a lentiviral vector expressing F12-Vif are protected from HIV-1 infection in an APOBEC3G-independent manner.

artículo científico publicado en 2005

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination

artículo científico publicado en 2013

The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene

artículo científico publicado en 2011

The Pharmacology of Gene and Cell Therapy.

artículo científico publicado en 2018

The future of gene therapy

artículo científico publicado en 2004

The recruitment of SOX/OCT complexes and the differential activity of HOXA1 and HOXB1 modulate the Hoxb1 auto-regulatory enhancer function

artículo científico publicado en 2001

The synthesis of HB Lepore Boston in peripheral blood

artículo científico publicado en 1979

Toward epidermal stem cell-mediated ex vivo gene therapy of junctional epidermolysis bullosa

artículo científico publicado en 2000

Towards a gene therapy clinical trial for epidermolysis bullosa

artículo científico publicado en 2006

Transcription factor binding sites are genetic determinants of retroviral integration in the human genome

artículo científico publicado en 2009

Transcriptional enhancers induce insertional gene deregulation independently from the vector type and design.

artículo científico publicado en 2009

Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment.

artículo científico publicado en 2016

Transduction of human hematopoietic stem cells by lentiviral vectors pseudotyped with the RD114-TR chimeric envelope glycoprotein

artículo científico publicado en 2007

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases

artículo científico publicado en 2013

Translocation and rearrangement of c-myc into immunoglobulin alpha heavy chain locus in primary cells from acute lymphocytic leukemia ⬇️

artículo científico publicado el 1 de septiembre de 1984

Two human homeobox genes, c1 and c8: structure analysis and expression in embryonic development

artículo científico publicado en 1987

beta Thalassemia associated with increased HB F production. Evidence for the existence of a heterocellular hereditary persistence of fetal hemoglobin (HPFH) determinant linked to beta thalassemia in a southern Italian population

artículo científico publicado en 1981

Lista de obras de Fulvio Mavilio

A human homoeo box gene specifically expressed in spinal cord during embryonic development

A new abnormal human hemoglobin: Hb Prato (alpha 2 31 (B12) Arg leads to Ser beta 2)

A new human hemoglobin variant: Hb BARI (alpha 2 45 (CD3) His leads to Gln beta 2)

A single epidermal stem cell strategy for safe ex vivo gene therapy.

Absence of an intrathecal immune reaction to a helper-dependent adenoviral vector delivered into the cerebrospinal fluid of non-human primates.

Alternative splicing caused by lentiviral integration in the human genome

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

C/EBPdelta regulates cell cycle and self-renewal of human limbal stem cells.

Characterization of Two Neuroblastoma Cell Lines Expressing Recombinant Nerve Growth Factor Receptors ⬇️

Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor

Comprehensive genomic access to vector integration in clinical gene therapy

Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.

Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.

Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted lentiviral vectors

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.

Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors

Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry.

Differential and stage-related expression in embryonic tissues of a new human homoeobox gene

Differential regulation by retinoic acid of the homeobox genes of the four HOX loci in human embryonal carcinoma cells ⬇️

Differential regulation of transferrin receptor gene expression in human hemopoietic cells: molecular and cellular aspects

Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation.

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.

Epithelial stem cells in corneal regeneration and epidermal gene therapy

Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors

Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in nonhuman primates.

Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair

Failure to correct murine muscular dystrophy

Functional dissection of a transcriptionally active, target-specific Hox-Pbx complex

Gene Therapy Approaches to Hemoglobinopathies.

Gene Therapy for Hemoglobinopathies

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study

Gene therapy approaches for epidermolysis bullosa

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Gene therapy in combination with tissue engineering to treat epidermolysis bullosa.

Gene therapy of inherited skin adhesion disorders

Gene therapy of inherited skin adhesion disorders: a critical overview.

Gene therapy of skin adhesion disorders (mini review).

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Gene therapy: back on track?

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency

Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.

Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells.

Genomic analysis of Sleeping Beauty transposon integration in human somatic cells

Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination

HOX gene activation by retinoic acid ⬇️

HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia

Haemoglobin Lepore Trait: Haematological and Structural Studies on the Italian Population ⬇️

Haemoglobin switching in human embryos: asynchrony of zeta----alpha and epsilon----gamma-globin switches in primitive and definite erythropoietic lineage

Hb J Baltimore (beta 16 (A13) Gly leads to Asp) in association with beta-thalassemia in a Sicilian family

Hemoglobin Gavello - alpha 2 beta 2 47 (CD6) Asp replaced by Gly. A new hemoglobin variant from Polesine (Italy)

Hemoglobin Legnano (alpha2 141 (HC3) Arg replaced by Leu beta2): a new abnormal human hemoglobin with high oxygen affinity

Hemoglobin bologna (alpha 2 beta 2 61 (E5) lys replaced by met). An abnormal human hemoglobin with low oxygen affinity

Heterocellular hereditary persistence of fetal hemoglobin (HPFH). Molecular mechanisms of abnormal ?-gene expression in association with ? thalassemia and linkage relationship with the ?-globin gene cluster

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.

High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors

Hot spots of retroviral integration in human CD34+ hematopoietic cells

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.

IL4 gene delivery to the CNS recruits regulatory T cells and induces clinical recovery in mouse models of multiple sclerosis.

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

Inhibition of retinoic acid-induced activation of 3' human HOXB genes by antisense oligonucleotides affects sequential activation of genes located upstream in the four HOX clusters.

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy.

Interactions between Retroviruses and the Host Cell Genome.

Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts

Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

Muscle regeneration by bone marrow-derived myogenic progenitors

Muscle-derived hematopoietic stem cells are hematopoietic in origin

Nuclear architecture dictates HIV-1 integration site selection.

Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication.

Occurrence of Hb J Paris in an Italian family and recombination studies on the free abnormal alpha-chain

Occurrence of Hb M Iwate (alpha 2 87 His leads to Tyr beta 2) in an Italian carrier