Zobrazeno 1 - 10
of 6 094
pro vyhledávání: '"DNA, Antisense"'
Akademický článek
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Autor:
Saoudi, Amel, Barberat, Sacha, le Coz, Olivier, Vacca, Ophélie, Doisy Caquant, Mathilde, Tensorer, Thomas, Sliwinski, Eric, Garcia, Luis, Muntoni, Francesco, Vaillend, Cyrille, Goyenvalle, Aurélie
Publikováno v:
In Molecular Therapy - Nucleic Acids 13 June 2023 32:173-188
Autor:
Amel Saoudi, Sacha Barberat, Olivier le Coz, Ophélie Vacca, Mathilde Doisy Caquant, Thomas Tensorer, Eric Sliwinski, Luis Garcia, Francesco Muntoni, Cyrille Vaillend, Aurélie Goyenvalle
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 32, Iss , Pp 173-188 (2023)
The mdx52 mouse model recapitulates a frequent mutation profile associated with brain involvement in Duchenne muscular dystrophy. Deletion of exon 52 impedes expression of two dystrophins (Dp427, Dp140) expressed in brain, and is eligible for therape
Externí odkaz:
https://doaj.org/article/14cbed576c444c01be01ae87e64766c9
Autor:
Sumiya K; Department of Chemistry and Biochemistry, University of Kitakyushu, 1-1 Hibikino, Wakamatsu-ku, Kitakyushu, Fukuoka 808-0135, Japan. Electronic address: c1dab001@eng.kitakyu-u.ac.jp., Izumi H; Department of Occupational Pneumology, Institute of Industrial Ecological Sciences, University of Occupational and Environmental Health, 1-1 Isegaoka, Yahatanishi-ku, Kitakyushu, Fukuoka 807-8555, Japan. Electronic address: h-izumi@med.uoeh-u.ac.jp., Sakurai K; Department of Chemistry and Biochemistry, University of Kitakyushu, 1-1 Hibikino, Wakamatsu-ku, Kitakyushu, Fukuoka 808-0135, Japan.
Publikováno v:
Bioorganic & medicinal chemistry letters [Bioorg Med Chem Lett] 2023 Oct 01; Vol. 94, pp. 129457. Date of Electronic Publication: 2023 Aug 25.
Autor:
Imbert M; U1179 INSERM, UFR des Sciences de la Santé-LIA BAHN CSM, Université de Versailles St-Quentin, Montigny le Bretonneux, France., Blandel F; U1179 INSERM, UFR des Sciences de la Santé-LIA BAHN CSM, Université de Versailles St-Quentin, Montigny le Bretonneux, France., Leumann C; Department of Chemistry and Biochemistry, University of Bern, Bern, Switzerland., Garcia L; U1179 INSERM, UFR des Sciences de la Santé-LIA BAHN CSM, Université de Versailles St-Quentin, Montigny le Bretonneux, France., Goyenvalle A; U1179 INSERM, UFR des Sciences de la Santé-LIA BAHN CSM, Université de Versailles St-Quentin, Montigny le Bretonneux, France.
Publikováno v:
Nucleic acid therapeutics [Nucleic Acid Ther] 2019 Oct; Vol. 29 (5), pp. 256-265. Date of Electronic Publication: 2019 Jun 11.
Autor:
Karima Relizani, Graziella Griffith, Lucía Echevarría, Faouzi Zarrouki, Patricia Facchinetti, Cyrille Vaillend, Christian Leumann, Luis Garcia, Aurélie Goyenvalle
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 8, Iss , Pp 144-157 (2017)
Antisense oligonucleotides (AONs) hold promise for therapeutic splice-switching correction in many genetic diseases. However, despite advances in AON chemistry and design, systemic use of AONs is limited due to poor tissue uptake and sufficient thera
Externí odkaz:
https://doaj.org/article/1d94d53beb4a47d8a7a1a68df902a18f
Autor:
Valérie Robin, Graziella Griffith, John-Paul L. Carter, Christian J. Leumann, Luis Garcia, Aurélie Goyenvalle
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 7, Iss C, Pp 81-89 (2017)
Spinal muscular atrophy (SMA) is a recessive disease caused by mutations in the SMN1 gene, which encodes the protein survival motor neuron (SMN), whose absence dramatically affects the survival of motor neurons. In humans, the severity of the disease
Externí odkaz:
https://doaj.org/article/1fd64706a1484ef79eea186b22c462a9
Akademický článek
Tento výsledek nelze pro nepřihlášené uživatele zobrazit.
K zobrazení výsledku je třeba se přihlásit.
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Autor:
Aghajan M; Department of Antisense Drug Discovery, IONIS Pharmaceuticals, 2855 Gazelle Court, Carlsbad, 92010, CA, USA., Guo S; Department of Antisense Drug Discovery, IONIS Pharmaceuticals, 2855 Gazelle Court, Carlsbad, 92010, CA, USA., Monia BP; Department of Antisense Drug Discovery, IONIS Pharmaceuticals, 2855 Gazelle Court, Carlsbad, 92010, CA, USA. bmonia@ionisph.com.
Publikováno v:
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2017; Vol. 1639, pp. 127-138.