Zobrazeno 1 - 10
of 12
pro vyhledávání: '"genetics [Dependovirus]"'
Autor:
Benjamin Stolte, Olivia Schreiber-Katz, René Günther, Claudia Diana Wurster, Susanne Petri, Alma Osmanovic, Maren Freigang, Zeljko Uzelac, Markus Leo, Otgonzul von Velsen, Wibke Bayer, Ulf Dittmer, Christoph Kleinschnitz, Tim Hagenacker
Publikováno v:
Human gene therapy 33, 968-976 (2022). doi:10.1089/hum.2022.054
5q-associated spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder that leads to progressive muscle atrophy and weakness. The disease is caused by a homozygous deletion or mutation in the survival of motor neuron 1 (SMN1) ge
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::68a249cf634b6e20bf7773fab7e41bb5
https://pub.dzne.de/record/165149
https://pub.dzne.de/record/165149
Autor:
Irena Tsui, Claudia B. Catarino, Piero Barboni, Günther Rudolph, Nancy J. Newman, Sara Silvestri, Alfredo A. Sadun, Michael Dattilo, Neringa Jurkute, Jean-François Girmens, Cosima Schertler, Magali Taiel, Rustum Karanjia, Claudia Priglinger, Maria K Gemenetzi, Armin Wolf, Manuela Contin, Jasmina Al-Tamami, Thomas Klopstock, James Acheson, Robert C. Sergott, Deborah Gibbs, Rabih Hage, Stephan R. Thurau, Adam A. DeBusk, Lidia Di Vito, Mark L. Moster, Valérie Biousse, Med Lindreth DuBois, Valerio Carelli, Gad Heilweil, Chiara La Morgia, Michele Carbonelli, Andrew Hendrick, Patrick Yu-Wai-Man, Jason H. Peragallo, Gerard Smits, Angelika Pressler, Martin Hildebrandt, Alcides Fernandes Filho, Michael Neuenhahn, Bettina von Livonius, Barrett Katz, Daniel R Muth, Siegfried G. Priglinger, Lauren Leitch-Devlin, Susan Mohamed, William R. Tucker, Maria Massini, Maria Eleftheriadou, Laure Blouin, Catherine Vignal-Clermont, Eman Hawy, Simona Degli Esposti, Heather Tollis, G. Baker Hubbard, Jannah Rutter Dobbs, José-Alain Sahel, Catherine Vignal, Melissa SantaMaria, Julie A. Haller
Publikováno v:
Ophthalmology: Journal of The American Academy of Ophthalmology
Ophthalmology: Journal of The American Academy of Ophthalmology, Elsevier, 2021, 128 (5), pp.649-660. ⟨10.1016/j.ophtha.2020.12.012⟩
Ophthalmology 128(5), 649-660 (2021). doi:10.1016/j.ophtha.2020.12.012
Ophthalmology: Journal of The American Academy of Ophthalmology, Elsevier, 2021, 128 (5), pp.649-660. ⟨10.1016/j.ophtha.2020.12.012⟩
Ophthalmology 128(5), 649-660 (2021). doi:10.1016/j.ophtha.2020.12.012
International audience; Purpose: To evaluate the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary optic neuropathy (LHON).Design: RESCUE is a multicenter, randomized, double-masked, sham-co
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::61639fdbd5cf15223e6676350bc8de96
https://hal.sorbonne-universite.fr/hal-03238444/file/1-s2.0-S0161642020311878-main.pdf
https://hal.sorbonne-universite.fr/hal-03238444/file/1-s2.0-S0161642020311878-main.pdf
Persistent repression of tau in the brain using engineered zinc finger protein transcription factors
Autor:
Susanne Wegmann, Bradley T. Hyman, Annemarie Ledeboer, Jeffrey C. Miller, Marta Perez-Rando, Kimberly Marlen, Richard T. Surosky, Bryan Zeitler, Caitlin Commins, Alison Chase, Michael C. Holmes, Sarah J. Hinkley, Alicia Goodwin, Riley N. Bannon, Lei Zhang, Brigit E. Riley, Hoang-Oanh B. Nguyen, Sarah L. DeVos, Edward J. Rebar, Ashley B. Robbins, Stephen Lam, Rainier Amora, Irina Ankoudinova, Qi Yu, Amy M. Pooler, Hung Tran, Nicholas A. Scarlott, Bianca T. Corjuc, Danny MacKenzie, Fyodor D. Urnov, H. Steve Zhang, Lisa Diez, Rachel E. Bennett
Publikováno v:
Science advances 7(12), eabe1611-(2021). doi:10.1126/sciadv.abe1611
Science Advances
Science Advances
Zinc finger proteins targeted to MAPT safely repress tau mRNA in the adult mouse brain and protect against Aβ toxicity.
Neuronal tau reduction confers resilience against β-amyloid and tau-related neurotoxicity in vitro and in vivo. Here, we in
Neuronal tau reduction confers resilience against β-amyloid and tau-related neurotoxicity in vitro and in vivo. Here, we in
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bb1e249560ae8a0539e415f467dba78e
Publikováno v:
Human gene therapy 32(13-14), 694-706 (2021). doi:10.1089/hum.2020.321
The most widely used vectors for gene delivery in the retina are recombinant adeno-associated virus (rAAV) vectors. They have proven to be safe and effective in retinal gene therapy studies aimed to treat inherited retinal dystrophies, although with
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2cd5b5edb63c35559d72c59fb4d525a9
Autor:
Günther Rudolph, Magali Taiel, Laure Blouin, Nancy J. Newman, Rustum Karanjia, Catherine Vignal-Clermont, Thomas Klopstock, Serge Picaud, Robert C. Sergott, Valérie Biousse, Gerard Smits, Caroline Chevalier, Mark L. Moster, Valerio Carelli, Chiara La Morgia, Alfredo A. Sadun, Harvey Masonson, Patrick Yu-Wai-Man, Barrett Katz, David J. Calkins, Yordak Salermo, Pierre Burguière, José-Alain Sahel
Publikováno v:
Science Translational Medicine
Science Translational Medicine, American Association for the Advancement of Science, 2020, 12 (573), ⟨10.1126/scitranslmed.aaz7423⟩
Science Translational Medicine, 2020, 12 (573), ⟨10.1126/scitranslmed.aaz7423⟩
Science translational medicine 12(573), eaaz7423 (2020). doi:10.1126/scitranslmed.aaz7423
Science Translational Medicine, American Association for the Advancement of Science, 2020, 12 (573), ⟨10.1126/scitranslmed.aaz7423⟩
Science Translational Medicine, 2020, 12 (573), ⟨10.1126/scitranslmed.aaz7423⟩
Science translational medicine 12(573), eaaz7423 (2020). doi:10.1126/scitranslmed.aaz7423
International audience; REVERSE is a randomized, double-masked, sham-controlled, multicenter, phase 3 clinical trial that evaluated the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary opti
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1e8b2819dfe0c1739c28bb816ac645e4
https://hal.sorbonne-universite.fr/hal-03373492/document
https://hal.sorbonne-universite.fr/hal-03373492/document
Publikováno v:
STAR Protocols 3(1), 101081 (2022). doi:10.1016/j.xpro.2021.101081
STAR Protocols
STAR Protocols, Vol 3, Iss 1, Pp 101081-(2022)
STAR Protocols
STAR Protocols, Vol 3, Iss 1, Pp 101081-(2022)
Summary This step-by-step protocol provides a fast and easy technique to label and/or genetically manipulate neural cells, achieved by intraventricular injection of viral vectors into neonatal mice under ultrasound guidance. Successful injection of a
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3d36e1f05377a562932e170270b44ef9
https://doi.org/10.1016/j.xpro.2021.101081
https://doi.org/10.1016/j.xpro.2021.101081
Autor:
Oliver Braganza, Milan Pabst, Johannes Niediek, Melike Bayraktar, Heinz Beck, Susanne Schoch, Florian Mormann, Holger Dannenberg
Publikováno v:
The journal of neuroscience 35(22), 8394-8410 (2015). doi:10.1523/JNEUROSCI.4460-14.2015
The medial septum/diagonal band of Broca complex (MSDB) is a key structure that modulates hippocampal rhythmogenesis. Cholinergic neurons of the MSDB play a central role in generating and pacing theta-band oscillations in the hippocampal formation du
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f613ccdc45e77d8f0aa56d1b0a199987
https://doi.org/10.1523/jneurosci.4460-14.2015
https://doi.org/10.1523/jneurosci.4460-14.2015
Autor:
Oskar Ortiz, Wolfgang Wurst, Ralf Kühn, Stefan Engelhardt, Karin Kühner, Stanislas Werfel, Dong-Jiunn Jeffery Truong
Publikováno v:
Nucleic Acids Research
Nucleic acids symposium series 43(13), 6450-6458 (2015). doi:10.1093/nar/gkv601
Nucleic Acids Res. 43, 6450-6458 (2015)
Nucleic acids symposium series 43(13), 6450-6458 (2015). doi:10.1093/nar/gkv601
Nucleic Acids Res. 43, 6450-6458 (2015)
Using CRISPR/Cas9, it is possible to target virtually any gene in any organism. A major limitation to its application in gene therapy is the size of Cas9 (>4 kb), impeding its efficient delivery via recombinant adeno-associated virus (rAAV). Therefor
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9f81a0268bf81c4a39a1e45b5731e435
http://europepmc.org/articles/PMC4513872
http://europepmc.org/articles/PMC4513872
Autor:
Aikaterini Nanou, Ke Ning, Pamela J. Shaw, Adrian Higginbottom, Matthew Wyles, Chiara F. Valori, Mimoun Azzouz
Publikováno v:
Molecular Therapy
Molecular therapy 21(8), 1486-1496 (2013). doi:10.1038/mt.2013.115
Molecular therapy 21(8), 1486-1496 (2013). doi:10.1038/mt.2013.115
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder with no effective treatment to date. Despite its multi-factorial aetiology, oxidative stress is hypothesized to be one of the key pathogenic mechanisms. It is thus propos
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c35cd0a1b6a940f57c306cec4b77b04b
https://doi.org/10.1038/mt.2013.115
https://doi.org/10.1038/mt.2013.115
Autor:
Chantal A. Mutsaers, Ellen J. Bennett, Basil Sharrack, Ke Ning, Chiara F. Valori, Pamela J. Shaw, Thomas H. Gillingwater, Daniel Little, Matthew Wyles, Mimoun Azzouz
Publikováno v:
Molecular therapy 23(2), 270-277 (2015). doi:10.1038/mt.2014.209
Molecular Therapy
Molecular Therapy
Spinal muscular atrophy (SMA) is the second most common genetic cause of death in childhood. However, no effective treatment is available to halt disease progression. SMA is caused by mutations in the survival motor neuron 1 (SMN1) gene. We previousl
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::12c6a58dc4d1cf5585d92ae8ca450a18