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Numrering	Referens	Omslagsbild	Hitta
1.	Kagiava, Alexia, et al. (författare) AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy. 2021 Ingår i: Gene therapy. - : Springer Science and Business Media LLC. - 1476-5462 .- 0969-7128. ; 28:10-11, s. 659-675 Tidskriftsartikel (refereegranskat)abstract Mutations in the GJB1 gene, encoding the gap junction (GJ) protein connexin32 (Cx32), cause X-linked Charcot-Marie-Tooth disease (CMT1X), an inherited demyelinating neuropathy. We developed a gene therapy approach for CMT1X using an AAV9 vector to deliver the GJB1/Cx32 gene under the myelin protein zero (Mpz) promoter for targeted expression in Schwann cells. Lumbar intrathecal injection of the AAV9-Mpz.GJB1 resulted in widespread biodistribution in the peripheral nervous system including lumbar roots, sciatic and femoral nerves, as well as in Cx32 expression in the paranodal non-compact myelin areas of myelinated fibers. A pre-, as well as post-onset treatment trial in Gjb1-null mice, demonstrated improved motor performance and sciatic nerve conduction velocities along with improved myelination and reduced inflammation in peripheral nerve tissues. Blood biomarker levels were also significantly ameliorated in treated mice. This study provides evidence that a clinically translatable AAV9-mediated gene therapy approach targeting Schwann cells could potentially treat CMT1X.
2.	Adams, Jennifer, et al. (författare) The Role of Science Education in a Changing World 2018 Rapport (övrigt vetenskapligt/konstnärligt)
3.	Christodoulou, John, et al. (författare) Heteronuclear NMR investigations of dynamic regions of intact Escherichia coli ribosomes. 2004 Ingår i: Proceedings of the National Academy of Sciences of the United States of America. - 0027-8424. ; 101:30, s. 10949-54 Tidskriftsartikel (refereegranskat)
4.	Henquell, Cecile, et al. (författare) Phylogenetic Patterns of Human Coxsackievirus B5 Arise from Population Dynamics between Two Genogroups and Reveal Evolutionary Factors of Molecular Adaptation and Transmission 2013 Ingår i: Journal of Virology. - 1098-5514. ; 87:22, s. 12249-12259 Tidskriftsartikel (refereegranskat)abstract The aim of this study was to gain insights into the tempo and mode of the evolutionary processes that sustain genetic diversity in coxsackievirus B5 (CVB5) and into the interplay with virus transmission. We estimated phylodynamic patterns with a large sample of virus strains collected in Europe by Bayesian statistical methods, reconstructed the ancestral states of genealogical nodes, and tested for selection. The genealogies estimated with the structural one-dimensional gene encoding the VP1 protein and nonstructural 3CD locus allowed the precise description of lineages over time and cocirculating virus populations within the two CVB5 clades, genogroups A and B. Strong negative selection shaped the evolution of both loci, but compelling phylogenetic data suggested that immune selection pressure resulted in the emergence of the two genogroups with opposed evolutionary pathways. The genogroups also differed in the temporal occurrence of the amino acid changes. The virus strains of genogroup A were characterized by sequential acquisition of nonsynonymous changes in residues exposed at the virus 5-fold axis. The genogroup B viruses were marked by selection of three changes in a different domain (VP1 C terminus) during its early emergence. These external changes resulted in a selective sweep, which was followed by an evolutionary stasis that is still ongoing after 50 years. The inferred population history of CVB5 showed an alternation of the prevailing genogroup during meningitis epidemics across Europe and is interpreted to be a consequence of partial cross-immunity.

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Resultat 1-4 av 4

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Typ av publikation: tidskriftsartikel (3); rapport (1)

Typ av innehåll: refereegranskat (3); övrigt vetenskapligt/konstnärligt (1)

Författare/redaktör: Richter, Jan (2); Christodoulou, Chris ... (2); Zetterberg, Henrik, ... (1); Dobson, Christopher ... (1); Robinson, Carol V (1); Larsson, Göran (1); visa fler...; Adams, Jennifer (1); Avraamidou, Lucy (1); Bayram-Jacobs, Dury (1); Boujaoude, Saouma (1); Bryan, Lynn (1); Christodoulou, Andri (1); Couso, Digna (1); Danielsson, Anna, Pr ... (1); Dillon, Justin (1); Erduran, Sibel (1); Evagorou, Maria (1); Goedhart, Martin (1); Kang, Nam-Hwa (1); Kaya, Ebru (1); Kayumova, Shakhnoza (1); Larsson, Johanna, 19 ... (1); Martin, Sonya (1); Martinez Chico, Mari ... (1); Marzabal, Ainoa (1); Savelsbergh, Elwin (1); Siry, Christina (1); van de Laar, Bart (1); Wals, Arjen (1); Wei, Bing (1); Wilmes, Sara (1); Zembal-Saul, Carla (1); Schleucher, Jurgen (1); Heslegrave, Amanda J (1); Böttiger, Blenda (1); Reilly, Mary M. (1); Christodoulou, John (1); Horvath, Rita (1); Fucini, Paola (1); Connell, Sean R (1); Pertinhez, Thelma A (1); Hanson, Charlotte L (1); Redfield, Christina (1); Nierhaus, Knud H (1); Henquell, Cecile (1); Mirand, Audrey (1); Schuffenecker, Isabe ... (1); Diedrich, Sabine (1); Terletskaia-Ladwig, ... (1); Peigue-Lafeuille, He ... (1); visa färre...

Lärosäte: Göteborgs universitet (1); Umeå universitet (1); Uppsala universitet (1); Lunds universitet (1)

Språk: Engelska (4)

Forskningsämne (UKÄ/SCB): Medicin och hälsovetenskap (2); Samhällsvetenskap (1)

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