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| 1 | Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction显示文摘In vivo genome editing represents a powerful strategy for both understanding basic biology and treating inherited diseases.However,it remains a challenge to develop universal and efficient in vivo genome-editing tools for tissues that comprise diverse cell types in either a dividing or non-dividing state.Here,we describe a versatile in vivo gene knock-in methodology that enables the targeting of a broad range of mutations and cell types through the insertion of a minigene at an intron of the target gene locus using an intracellularly linearized single homology arm donor.As a proof-of-concept,we focused on a mouse model of prematureaging caused by a dominant point mutation,which is difficult to repair using existing in vivo genome-editing tools.Systemic treatment using our new method ameliorated aging-associated phenotypes and extended animal lifespan,thus highlighting the potential of this methodology for a broad range of in vivo genome-editing applications. | Keiichiro Suzuki Mako Yamamoto Reyna Hernandez-Benitez Zhe Li Christopher Wei Rupa Devi Soligalla Emi Aizawa Fumiyuki Hatanaka Masakazu Kurita Pradeep Reddy Alejandro Ocampo Tomoaki Hishida Masahiro Sakurai Amy NNemeth Estrella Nunez Delicado Josep MCampistol Pierre Magistretti Pedro Guillen Concepcion Rodriguez Esteban Jianhui Gong Yilin Yuan Ying Gu Guang-Hui Liu Carlos Lopez-Otin Jun Wu Kun Zhang Juan Carlos Izpisua Belmonte | 2019 | Cell Research2019，29，10： | 2 |
| 2 | Metabolic coupling between glia and neurons显示文摘 | Tsacopoulos M Magistretti P J | 1996 | J Neurosci1996，16，： | 1 |
| 3 | Aquaporins in brain : distribution, physiology, and pathophysiology显示文摘 | Badaut J Lasbennes F Magistretti PJ | 2002 | J Cereb Blood Flow Metab2002，22，4： | 1 |
| 4 | Aquaporins in brain: distribution, physiology, and pathophysiology显示文摘 | Badaut J Lasbennes F Magistretti PJ | 2002 | J Cereb Blood Flow Metab2002，22，： | 1 |
| 5 | Comparative bioavailability of silipide, a new flavanolignan complex, in rats 显示文摘 | Magistretti M J Giachetti C | 1992 | Eur J Drug Metab Pharmacokinet1992，17，1： | 1 |
| 6 | Digital holographic microscopy: a noninvasive contrast imaging technique allowing quantitative visualization of living cells with sub-wavelength axial accuracy显示文摘 | P Marquet B Rappaz P J Magistretti | 2005 | Opt Lett2005，30，5： | 1 |
| 7 | Transrectal real-time elastography of the prostate : normal patterns 显示文摘 | Goddi A Sacchi A Magistretti G | 2011 | J Ultrasound2011，14，4： | 1 |
| 8 | Cerebral metabolic effects of exogenous lactate supplementation on the injured human brain显示文摘 | Pierre Bouzat Nathalie Sala Tamarah Suys Jean-Baptiste Zerlauth Pedro Marques-Vidal Fran?ois Feihl Jocelyne Bloch Mahmoud Messerer Marc Levivier Reto Meuli Pierre J. Magistretti Mauro Oddo | 2014 | Intensive Care Medicine2014，，3： | 1 |
| 9 | Aquaporins in brain:distribution,physiology,and pathophysiology显示文摘 | Badaut J Lasbennes F Magistretti P J | 2002 | J Cereb Blood Flow Metab2002，22，4： | 1 |
| 10 | Arteritis following intra-arterial chemotherapy for liver tumors显示文摘 | Belli L Magistretti G Puricelli GP | 1997 | Eur Radiol1997，7，： | 1 |
| 11 | Aquaporins in brain:distributions, Physiology, and pathophysiology显示文摘 | Badaut J Lasbennes F Magistretti PJ | 2002 | J Cereb Blood Flow Metab2002，22，4： | 1 |
| 12 | Energy on demand显示文摘 | MAGISTRETTI PJ PELLERIN L ROTHMAN DL | 1999 | J Science1999，283，： | 1 |
| 13 | Comparative bioavailability of Silipide, a new flavano- lignan complex, in rats 显示文摘 | Morazzoni P Magistretti M J Giachetti C | 1992 | Eur J Drug Metab Pharmacokinet1992，17，1： | 1 |
| 14 | Aquaporin in brain: Distribution, Physiology, and Pathophysiology 显示文摘 | Badaut J Lasbennes F Magistretti P J | 2002 | J Cereb Blood Flow Metab2002，22，： | 1 |
| 15 | Smart mobs for urban monitoring with a vehicular sensDr network显示文摘 | Magistretti E Lee U Gerla M | 2006 | IEEE Wireless Comm2006，13，5： | 1 |
| 16 | MCT2 is a major neuronal monocarboxylate transporter in the adult mouse brain 显示文摘 | Pierre K Magistretti P J Pellerin L | 2002 | Cereb Blood Flow Metab2002，22，5： | 1 |
| 17 | Aquaporins in brain:distributions,Physiology,and pathophysiology显示文摘 | Badaut J Lasbennes F Magistretti PJ | 2002 | International Society of Cerebral Blood Flow and Metabolism2002，22，4： | 1 |
| 18 | New evidence of neuroprotec- tion by lactate aftertransient focal cerebral ischaemia: extended benefit after injection and efficacy of intravenous drain- istration显示文摘 | Berthet C Castillo X Magistretti PJ | 2012 | Cerebrovasc Dis2012，34，56： | 1 |
| 19 | Aquaporins in brain: distribution, physiology, and pathophysiology 显示文摘 | Badaut J Lasbennes F Magistretti PJ | 2002 | J Cereb Blood Flow Metab2002，22，4： | 1 |
| 20 | Metabolic coup ling glia and neurons显示文摘 | Tsacopulo M Magistretti PJ | 1996 | J Neuroxci1996，16，8： | 1 |
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