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Sökning: onr:"swepub:oai:lup.lub.lu.se:8f1ca54b-0068-4f27-be30-8c56998048b3" > Combination of cGMP...

LIBRIS Formathandbok  (Information om MARC21)
FältnamnIndikatorerMetadata
00004825naa a2200601 4500
001oai:lup.lub.lu.se:8f1ca54b-0068-4f27-be30-8c56998048b3
003SwePub
008180523s2018 | |||||||||||000 ||eng|
0247 a https://lup.lub.lu.se/record/8f1ca54b-0068-4f27-be30-8c56998048b32 URI
0247 a https://doi.org/10.1073/pnas.17187921152 DOI
040 a (SwePub)lu
041 a engb eng
042 9 SwePub
072 7a art2 swepub-publicationtype
072 7a ref2 swepub-contenttype
1001 a Vighi, Eleonorau University of Modena and Reggio Emilia4 aut
2451 0a Combination of cGMP analogue and drug delivery system provides functional protection in hereditary retinal degeneration
264 c 2018-03-12
264 1b 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 7a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Oftalmologi0 (SwePub)302172 hsv//swe
650 7a 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
7001 a Trifunovic, Draganau University of Tübingen4 aut
7001 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
7001 a Rentsch, Andreasu BIOLOG Life Science Institute4 aut
7001 a Hoffmann, Doritu University of Modena and Reggio Emilia4 aut
7001 a Sahaboglu, Ayseu University of Tübingen4 aut
7001 a Strasser, Torstenu University of Tübingen4 aut
7001 a Kulkarni, Manoju University of Tübingen4 aut
7001 a Bertolotti, Evelinau University of Modena and Reggio Emilia4 aut
7001 a Van Den Heuvel, Angeliqueu to-BBB technologies BV4 aut
7001 a Peters, Tobiasu University of Tübingen4 aut
7001 a Reijerkerk, Arieu to-BBB technologies BV4 aut
7001 a Euler, Thomasu University of Tübingen4 aut
7001 a Ueffing, Mariusu University of Tübingen4 aut
7001 a Schwede, Franku BIOLOG Life Science Institute4 aut
7001 a Genieser, Hans Gottfriedu BIOLOG Life Science Institute4 aut
7001 a Gaillard, Pieteru to-BBB technologies BV4 aut
7001 a Marigo, Valeriau University of Modena and Reggio Emilia4 aut
7001 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
7001 a Paquet-Durand, Françoisu University of Tübingen4 aut0 (Swepub:lu)oft-fpa
7102 a University of Modena and Reggio Emiliab University of Tübingen4 org
7730 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
7730 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
8564 u http://dx.doi.org/10.1073/pnas.1718792115y FULLTEXT
8564 u https://www.pnas.org/content/pnas/115/13/E2997.full.pdf
8564 8u https://lup.lub.lu.se/record/8f1ca54b-0068-4f27-be30-8c56998048b3
8564 8u https://doi.org/10.1073/pnas.1718792115

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