Sökning: onr:"swepub:oai:lup.lub.lu.se:8f1ca54b-0068-4f27-be30-8c56998048b3" > Combination of cGMP...
Fältnamn | Indikatorer | Metadata |
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000 | 04825naa a2200601 4500 | |
001 | oai:lup.lub.lu.se:8f1ca54b-0068-4f27-be30-8c56998048b3 | |
003 | SwePub | |
008 | 180523s2018 | |||||||||||000 ||eng| | |
024 | 7 | a https://lup.lub.lu.se/record/8f1ca54b-0068-4f27-be30-8c56998048b32 URI |
024 | 7 | a https://doi.org/10.1073/pnas.17187921152 DOI |
040 | a (SwePub)lu | |
041 | a engb eng | |
042 | 9 SwePub | |
072 | 7 | a art2 swepub-publicationtype |
072 | 7 | a ref2 swepub-contenttype |
100 | 1 | a Vighi, Eleonorau University of Modena and Reggio Emilia4 aut |
245 | 1 0 | a Combination of cGMP analogue and drug delivery system provides functional protection in hereditary retinal degeneration |
264 | c 2018-03-12 | |
264 | 1 | b Proceedings of the National Academy of Sciences,c 2018 |
520 | a Inherited retinal degeneration (RD) is a devastating and currently untreatable neurodegenerative condition that leads to loss of photoreceptor cells and blindness. The vast genetic heterogeneity of RD, the lack of “druggable” targets, and the access-limiting blood–retinal barrier (BRB) present major hurdles toward effective therapy development. Here, we address these challenges (i) by targeting cGMP (cyclic guanosine- 3′,5′-monophosphate) signaling, a disease driver common to different types of RD, and (ii) by combining inhibitory cGMP analogs with a nanosized liposomal drug delivery system designed to facilitate transport across the BRB. Based on a screen of several cGMP analogs we identified an inhibitory cGMP analog that interferes with activation of photoreceptor cell death pathways. Moreover, we found liposomal encapsulation of the analog to achieve efficient drug targeting to the neuroretina. This pharmacological treatment markedly preserved in vivo retinal function and counteracted photoreceptor degeneration in three different in vivo RD models. Taken together, we show that a defined class of compounds for RD treatment in combination with an innovative drug delivery method may enable a single type of treatment to address genetically divergent RD-type diseases. | |
650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Oftalmologi0 (SwePub)302172 hsv//swe |
650 | 7 | a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Ophthalmology0 (SwePub)302172 hsv//eng |
653 | a Apoptosis | |
653 | a Calpain | |
653 | a CNG channel | |
653 | a In vivo imaging | |
653 | a PKG | |
700 | 1 | a Trifunovic, Draganau University of Tübingen4 aut |
700 | 1 | a Veiga-Crespo, Patriciau Lund University,Lunds universitet,Retinal degeneration: Molekylär patologi,Forskargrupper vid Lunds universitet,Retinal degeneration: Molecular Pathology,Lund University Research Groups4 aut0 (Swepub:lu)med-pve |
700 | 1 | a Rentsch, Andreasu BIOLOG Life Science Institute4 aut |
700 | 1 | a Hoffmann, Doritu University of Modena and Reggio Emilia4 aut |
700 | 1 | a Sahaboglu, Ayseu University of Tübingen4 aut |
700 | 1 | a Strasser, Torstenu University of Tübingen4 aut |
700 | 1 | a Kulkarni, Manoju University of Tübingen4 aut |
700 | 1 | a Bertolotti, Evelinau University of Modena and Reggio Emilia4 aut |
700 | 1 | a Van Den Heuvel, Angeliqueu to-BBB technologies BV4 aut |
700 | 1 | a Peters, Tobiasu University of Tübingen4 aut |
700 | 1 | a Reijerkerk, Arieu to-BBB technologies BV4 aut |
700 | 1 | a Euler, Thomasu University of Tübingen4 aut |
700 | 1 | a Ueffing, Mariusu University of Tübingen4 aut |
700 | 1 | a Schwede, Franku BIOLOG Life Science Institute4 aut |
700 | 1 | a Genieser, Hans Gottfriedu BIOLOG Life Science Institute4 aut |
700 | 1 | a Gaillard, Pieteru to-BBB technologies BV4 aut |
700 | 1 | a Marigo, Valeriau University of Modena and Reggio Emilia4 aut |
700 | 1 | a Ekström, Peru Lund University,Lunds universitet,Retinal degeneration: Molekylär patologi,Forskargrupper vid Lunds universitet,Retinal degeneration: Molecular Pathology,Lund University Research Groups4 aut0 (Swepub:lu)zoof-pek |
700 | 1 | a Paquet-Durand, Françoisu University of Tübingen4 aut0 (Swepub:lu)oft-fpa |
710 | 2 | a University of Modena and Reggio Emiliab University of Tübingen4 org |
773 | 0 | t Proceedings of the National Academy of Sciences of the United States of Americad : Proceedings of the National Academy of Sciencesg 115:13, s. 2997-3006q 115:13<2997-3006x 0027-8424 |
773 | 0 | t Proceedings of the National Academy of Sciencesd : Proceedings of the National Academy of Sciencesg 115:13, s. 2997-3006q 115:13<2997-3006x 1091-6490 |
856 | 4 | u http://dx.doi.org/10.1073/pnas.1718792115y FULLTEXT |
856 | 4 | u https://www.pnas.org/content/pnas/115/13/E2997.full.pdf |
856 | 4 8 | u https://lup.lub.lu.se/record/8f1ca54b-0068-4f27-be30-8c56998048b3 |
856 | 4 8 | u https://doi.org/10.1073/pnas.1718792115 |
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