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  1. Article ; Online: Viral gene therapy for paediatric neurological diseases: progress to clinical reality.

    Privolizzi, Riccardo / Chu, Wing Sum / Tijani, Maha / Ng, Joanne

    Developmental medicine and child neurology

    2021  Volume 63, Issue 9, Page(s) 1019–1029

    Abstract: In the era of genomic medicine, diagnoses of rare paediatric neurological diseases are increasing. Many are untreatable and life-limiting, leading to an exceptional increase in gene therapy development. It is estimated that 20 gene therapy products will ... ...

    Abstract In the era of genomic medicine, diagnoses of rare paediatric neurological diseases are increasing. Many are untreatable and life-limiting, leading to an exceptional increase in gene therapy development. It is estimated that 20 gene therapy products will have received approval from the US Food and Drug Administration by 2025. With viral gene therapy considered a potential single-dose cure for patients with spinal muscular atrophy type 1 as one example, and contemporaneously tragically resulting in the deaths of three male children with X-linked myotubular myopathy receiving high-dose gene therapy in 2020, what is the current state of gene therapy? What is behind the decades of hype around viral gene therapy and is it high impact, but high risk? In this review, we outline principles of viral gene therapy development and summarize the most recent clinical evidence for the therapeutic effect of gene therapy in paediatric neurological diseases. We discuss adeno-associated virus and lentiviral vectors, antisense oligonucleotides, emerging genetic editing approaches, and current limitations that the field still faces. What this paper adds Viral gene therapy development and clinically used transgenes, regulatory elements, capsids, dosage, and delivery routes are summarized. Viral gene therapy for 18 childhood neurological disorders involving over 600 children in 40 clinical trials are reviewed.
    MeSH term(s) Clinical Trials as Topic ; Dependovirus/genetics ; Genetic Diseases, Inborn/therapy ; Genetic Therapy/methods ; Genetic Vectors ; Humans ; Lentivirus/genetics ; Nervous System Diseases/genetics ; Nervous System Diseases/therapy ; Transduction, Genetic/methods ; Transfection/methods
    Language English
    Publishing date 2021-04-09
    Publishing country England
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Review
    ZDB-ID 80369-8
    ISSN 1469-8749 ; 0012-1622
    ISSN (online) 1469-8749
    ISSN 0012-1622
    DOI 10.1111/dmcn.14885
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

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  2. Article ; Online: Characterization of Antibody Interactions with the G Protein of Vesicular Stomatitis Virus Indiana Strain and Other Vesiculovirus G Proteins.

    Munis, Altar M / Tijani, Maha / Hassall, Mark / Mattiuzzo, Giada / Collins, Mary K / Takeuchi, Yasuhiro

    Journal of virology

    2018  Volume 92, Issue 23

    Abstract: Vesicular stomatitis virus Indiana strain G protein (VSVind.G) is the most commonly used envelope glycoprotein to pseudotype lentiviral vectors (LV) for experimental and clinical applications. Recently, G proteins derived from other vesiculoviruses (VesG) ...

    Abstract Vesicular stomatitis virus Indiana strain G protein (VSVind.G) is the most commonly used envelope glycoprotein to pseudotype lentiviral vectors (LV) for experimental and clinical applications. Recently, G proteins derived from other vesiculoviruses (VesG), for example, Cocal virus, have been proposed as alternative LV envelopes with possible advantages over VSVind.G. Well-characterized antibodies that recognize VesG will be useful for vesiculovirus research, development of G protein-containing advanced therapy medicinal products (ATMPs), and deployment of VSVind-based vaccine vectors. Here, we show that one commercially available monoclonal antibody, 8G5F11, binds to and neutralizes G proteins from three strains of VSV, as well as Cocal and Maraba viruses, whereas the other commercially available monoclonal anti-VSVind.G antibody, IE9F9, binds to and neutralizes only VSVind.G. Using a combination of G protein chimeras and site-directed mutations, we mapped the binding epitopes of IE9F9 and 8G5F11 on VSVind.G. IE9F9 binds close to the receptor binding site and competes with soluble low-density lipoprotein receptor (LDLR) for binding to VSVind.G, explaining its mechanism of neutralization. In contrast, 8G5F11 binds close to a region known to undergo conformational changes when the G protein moves to its postfusion structure, and we propose that 8G5F11 cross-neutralizes VesGs by inhibiting this.
    MeSH term(s) Amino Acid Sequence ; Antibodies, Monoclonal/immunology ; Antibodies, Monoclonal/metabolism ; Antibodies, Neutralizing/immunology ; Antibodies, Neutralizing/metabolism ; Antigens, Viral/genetics ; Antigens, Viral/immunology ; Antigens, Viral/metabolism ; Cross Reactions ; Epitopes/immunology ; Epitopes/metabolism ; HEK293 Cells ; Humans ; Membrane Glycoproteins/genetics ; Membrane Glycoproteins/immunology ; Membrane Glycoproteins/metabolism ; Neutralization Tests ; Phylogeny ; Sequence Homology ; Vesicular Stomatitis/immunology ; Vesicular Stomatitis/metabolism ; Vesicular Stomatitis/virology ; Vesicular stomatitis Indiana virus/immunology ; Viral Envelope Proteins/genetics ; Viral Envelope Proteins/immunology ; Viral Envelope Proteins/metabolism
    Chemical Substances Antibodies, Monoclonal ; Antibodies, Neutralizing ; Antigens, Viral ; Epitopes ; G protein, vesicular stomatitis virus ; Membrane Glycoproteins ; Viral Envelope Proteins
    Language English
    Publishing date 2018-11-12
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 80174-4
    ISSN 1098-5514 ; 0022-538X
    ISSN (online) 1098-5514
    ISSN 0022-538X
    DOI 10.1128/JVI.00900-18
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

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    Jg. 1995 - 2021: Lesesall (1.OG)
    ab Jg. 2022: Lesesaal (EG)

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  3. Article ; Online: TMEM16F activation by Ca

    Bricogne, Christopher / Fine, Michael / Pereira, Pedro M / Sung, Julia / Tijani, Maha / Wang, Youxue / Henriques, Ricardo / Collins, Mary K / Hilgemann, Donald W

    Scientific reports

    2019  Volume 9, Issue 1, Page(s) 619

    Abstract: TMEM16F is a ... ...

    Abstract TMEM16F is a Ca
    MeSH term(s) Anoctamins/genetics ; Anoctamins/metabolism ; CRISPR-Cas Systems/genetics ; Calcium/metabolism ; Cell Line ; Cell Membrane/metabolism ; Flow Cytometry ; Humans ; Jurkat Cells ; Lentivirus/genetics ; Microscopy, Confocal ; Phospholipid Transfer Proteins/genetics ; Phospholipid Transfer Proteins/metabolism ; Programmed Cell Death 1 Receptor/genetics ; Programmed Cell Death 1 Receptor/metabolism
    Chemical Substances ANO6 protein, human ; Anoctamins ; Phospholipid Transfer Proteins ; Programmed Cell Death 1 Receptor ; Calcium (SY7Q814VUP)
    Language English
    Publishing date 2019-01-24
    Publishing country England
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ZDB-ID 2615211-3
    ISSN 2045-2322 ; 2045-2322
    ISSN (online) 2045-2322
    ISSN 2045-2322
    DOI 10.1038/s41598-018-37056-x
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: Author Correction: TMEM16F activation by Ca

    Bricogne, Christopher / Fine, Michael / Pereira, Pedro M / Sung, Julia / Tijani, Maha / Wang, Youxue / Henriques, Ricardo / Collins, Mary K / Hilgemann, Donald W

    Scientific reports

    2019  Volume 9, Issue 1, Page(s) 7705

    Abstract: A correction to this article has been published and is linked from the HTML and PDF versions of this paper. The error has been fixed in the paper. ...

    Abstract A correction to this article has been published and is linked from the HTML and PDF versions of this paper. The error has been fixed in the paper.
    Language English
    Publishing date 2019-05-17
    Publishing country England
    Document type Published Erratum
    ZDB-ID 2615211-3
    ISSN 2045-2322 ; 2045-2322
    ISSN (online) 2045-2322
    ISSN 2045-2322
    DOI 10.1038/s41598-019-43808-0
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article ; Online: Cell-cell adhesion regulates Merlin/NF2 interaction with the PAF complex.

    Roehrig, Anne E / Klupsch, Kristina / Oses-Prieto, Juan A / Chaib, Selim / Henderson, Stephen / Emmett, Warren / Young, Lucy C / Surinova, Silvia / Blees, Andreas / Pfeiffer, Anett / Tijani, Maha / Brunk, Fabian / Hartig, Nicole / Muñoz-Alegre, Marta / Hergovich, Alexander / Jennings, Barbara H / Burlingame, Alma L / Rodriguez-Viciana, Pablo

    PloS one

    2021  Volume 16, Issue 8, Page(s) e0254697

    Abstract: The PAF complex (PAFC) coordinates transcription elongation and mRNA processing and its CDC73/parafibromin subunit functions as a tumour suppressor. The NF2/Merlin tumour suppressor functions both at the cell cortex and nucleus and is a key mediator of ... ...

    Abstract The PAF complex (PAFC) coordinates transcription elongation and mRNA processing and its CDC73/parafibromin subunit functions as a tumour suppressor. The NF2/Merlin tumour suppressor functions both at the cell cortex and nucleus and is a key mediator of contact inhibition but the molecular mechanisms remain unclear. In this study we have used affinity proteomics to identify novel Merlin interacting proteins and show that Merlin forms a complex with multiple proteins involved in RNA processing including the PAFC and the CHD1 chromatin remodeller. Tumour-derived inactivating mutations in both Merlin and the CDC73 PAFC subunit mutually disrupt their interaction and growth suppression by Merlin requires CDC73. Merlin interacts with the PAFC in a cell density-dependent manner and we identify a role for FAT cadherins in regulating the Merlin-PAFC interaction. Our results suggest that in addition to its function within the Hippo pathway, Merlin is part of a tumour suppressor network regulated by cell-cell adhesion which coordinates post-initiation steps of the transcription cycle of genes mediating contact inhibition.
    MeSH term(s) Cell Adhesion/genetics ; Cell Proliferation/genetics ; Chromatin/genetics ; Chromatin Assembly and Disassembly/genetics ; Contact Inhibition/genetics ; DNA Helicases/genetics ; DNA-Binding Proteins/genetics ; Gene Expression Regulation/genetics ; HEK293 Cells ; Humans ; Neoplasms/genetics ; Neoplasms/pathology ; Neurofibromin 2/genetics ; Protein Binding/genetics ; Protein Interaction Maps/genetics ; Signal Transduction/genetics ; Tumor Suppressor Proteins/genetics
    Chemical Substances CDC73 protein, human ; Chromatin ; DNA-Binding Proteins ; Neurofibromin 2 ; Tumor Suppressor Proteins ; DNA Helicases (EC 3.6.4.-) ; CHD1 protein, human (EC 3.6.4.12)
    Language English
    Publishing date 2021-08-23
    Publishing country United States
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Research Support, Non-U.S. Gov't
    ZDB-ID 2267670-3
    ISSN 1932-6203 ; 1932-6203
    ISSN (online) 1932-6203
    ISSN 1932-6203
    DOI 10.1371/journal.pone.0254697
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article: Lentivector Producer Cell Lines with Stably Expressed Vesiculovirus Envelopes.

    Tijani, Maha / Munis, Altar M / Perry, Christopher / Sanber, Khaled / Ferraresso, Marta / Mukhopadhyay, Tarit / Themis, Michael / Nisoli, Ilaria / Mattiuzzo, Giada / Collins, Mary K / Takeuchi, Yasuhiro

    Molecular therapy. Methods & clinical development

    2018  Volume 10, Page(s) 303–312

    Abstract: Retroviral and lentiviral vectors often use the envelope G protein from the vesicular stomatitis virus Indiana strain (VSVind.G). However, lentivector producer cell lines that stably express VSVind.G have not been reported, presumably because of its ... ...

    Abstract Retroviral and lentiviral vectors often use the envelope G protein from the vesicular stomatitis virus Indiana strain (VSVind.G). However, lentivector producer cell lines that stably express VSVind.G have not been reported, presumably because of its cytotoxicity, preventing simple scale-up of vector production. Interestingly, we showed that VSVind.G and other vesiculovirus G from the VSV New Jersey strain (VSVnj), Cocal virus (COCV), and Piry virus (PIRYV) could be constitutively expressed and supported lentivector production for up to 10 weeks. All G-enveloped particles were robust, allowing concentration and freeze-thawing. COCV.G and PIRYV.G were resistant to complement inactivation, and, using chimeras between VSVind.G and COCV.G, the determinant for complement inactivation of VSVind.G was mapped to amino acid residues 136-370. Clonal packaging cell lines using COCV.G could be generated; however, during attempts to establish LV producer cells, vector superinfection was observed following the introduction of a lentivector genome. This could be prevented by culturing the cells with the antiviral drug nevirapine. As an alternative countermeasure, we demonstrated that functional lentivectors could be reconstituted by admixing supernatant from stable cells producing unenveloped virus with supernatant containing envelopes harvested from cells stably expressing VSVind.G, COCV.G, or PIRYV.G.
    Language English
    Publishing date 2018-08-07
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2872938-9
    ISSN 2329-0501 ; 2329-0501
    ISSN (online) 2329-0501
    ISSN 2329-0501
    DOI 10.1016/j.omtm.2018.07.013
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 7107: Show issues Location:
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  7. Article ; Online: Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.

    Karda, Rajvinder / Rahim, Ahad A / Wong, Andrew M S / Suff, Natalie / Diaz, Juan Antinao / Perocheau, Dany P / Tijani, Maha / Ng, Joanne / Baruteau, Julien / Martin, Nuria Palomar / Hughes, Michael / Delhove, Juliette M K M / Counsell, John R / Cooper, Jonathan D / Henckaerts, Els / Mckay, Tristan R / Buckley, Suzanne M K / Waddington, Simon N

    Scientific reports

    2020  Volume 10, Issue 1, Page(s) 2121

    Abstract: We have previously designed a library of lentiviral vectors to generate somatic-transgenic rodents to monitor signalling pathways in diseased organs using whole-body bioluminescence imaging, in conscious, freely moving rodents. We have now expanded this ... ...

    Abstract We have previously designed a library of lentiviral vectors to generate somatic-transgenic rodents to monitor signalling pathways in diseased organs using whole-body bioluminescence imaging, in conscious, freely moving rodents. We have now expanded this technology to adeno-associated viral vectors. We first explored bio-distribution by assessing GFP expression after neonatal intravenous delivery of AAV8. We observed widespread gene expression in, central and peripheral nervous system, liver, kidney and skeletal muscle. Next, we selected a constitutive SFFV promoter and NFκB binding sequence for bioluminescence and biosensor evaluation. An intravenous injection of AAV8 containing firefly luciferase and eGFP under transcriptional control of either element resulted in strong and persistent widespread luciferase expression. A single dose of LPS-induced a 10-fold increase in luciferase expression in AAV8-NFκB mice and immunohistochemistry revealed GFP expression in cells of astrocytic and neuronal morphology. Importantly, whole-body bioluminescence persisted up to 240 days. We have validated a novel biosensor technology in an AAV system by using an NFκB response element and revealed its potential to monitor signalling pathway in a non-invasive manner in a model of LPS-induced inflammation. This technology complements existing germline-transgenic models and may be applicable to other rodent disease models.
    MeSH term(s) Animals ; Biosensing Techniques/methods ; Dependovirus/genetics ; Gene Expression/genetics ; Genetic Vectors/genetics ; Green Fluorescent Proteins/genetics ; Inflammation/genetics ; Luciferases, Firefly/genetics ; Mice ; Mice, Transgenic/genetics ; NF-kappa B/genetics ; Promoter Regions, Genetic/genetics ; Signal Transduction/genetics ; Spleen Focus-Forming Viruses/genetics ; Transcription, Genetic/genetics
    Chemical Substances NF-kappa B ; Green Fluorescent Proteins (147336-22-9) ; Luciferases, Firefly (EC 1.13.12.7)
    Language English
    Publishing date 2020-02-07
    Publishing country England
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 2615211-3
    ISSN 2045-2322 ; 2045-2322
    ISSN (online) 2045-2322
    ISSN 2045-2322
    DOI 10.1038/s41598-020-59075-3
    Database MEDical Literature Analysis and Retrieval System OnLINE

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