Merve Sen
University of Tübingen
TransgeneGeneGenetic enhancementGene silencingTranslation (biology)High-content screeningEndoplasmic reticulumEndoplasmic-reticulum-associated protein degradationMagnetofectionChemistryRhodopsinIn vitroAutophagyIn vivoSmall interfering RNAEx vivoProteasomeRetinaEthylene glycolDrug deliveryDrugInterference (genetic)RetinalRetinal degenerationRetinitis pigmentosaProteostasisPhotoreceptor degenerationRetinal DisorderPharmacological modulationChaperone (protein)MutationHomeostasisRNAMedicineCell biologyCytosol
8Publications
2H-index
5Citations
Publications 9
Newest
#1Merve Sen (University of Tübingen)H-Index: 2
#2Al-Amin (UNIPD: University of Padua)H-Index: 1
Last. Marius Ueffing (University of Tübingen)H-Index: 72
view all 10 authors...
Abstract null null Mutations in rhodopsin lead to its misfolding resulting in autosomal dominant retinitis pigmentosa (adRP). Pharmacological inhibition of the ATP-driven chaperone valosin-containing protein (VCP), a molecular checkpoint for protein quality control, slows down retinal degeneration in animal models. However, poor water-solubility of VCP inhibitors poses a challenge to their clinical translation as intravitreal injections for retinal treatment. In order to enable the delivery of V...
Source
#1Merve Sen (University of Tübingen)H-Index: 2
#2Oksana Kutsyr (University of Alicante)H-Index: 4
Last. Marius Ueffing (University of Tübingen)H-Index: 72
view all 6 authors...
1.Rhodopsin (RHO) misfolding mutations are a common cause of the blinding disease autosomal dominant retinitis pigmentosa (adRP). The most prevalent mutation, RHOP23H, results in its misfolding and retention in the Endoplasmic Reticulum (ER). Under homeostatic conditions, misfolded proteins are selectively identified, retained at the ER, and cleared via ER-associated degradation (ERAD) and/or autophagy. Overload of these degradation processes for a prolonged period leads to imbalanced proteostas...
1 CitationsSource
#1Merve Sen (University of Tübingen)H-Index: 2
#2Al-Amin M (UNIPD: University of Padua)
Last. Marius Ueffing (University of Tübingen)H-Index: 72
view all 10 authors...
1.Mutations in rhodopsin lead to its misfolding resulting in autosomal dominant retinitis pigmentosa (adRP). Pharmacological inhibition of the ATP-driven chaperone valosin- containing protein (VCP), a molecular checkpoint for protein quality control, slows down retinal degeneration in animal models. However, poor water-solubility of VCP inhibitors poses a challenge to their clinical translation as intravitreal injections for retinal treatment. In order to enable the delivery of VCP inhibitors, w...
Source
#1Marco BassettoH-Index: 1
#2Merve Sen (University of Tübingen)H-Index: 2
Last. Olivier ZelphatiH-Index: 14
view all 8 authors...
The prevalence of retinal disorders associated with visual impairment and blindness is increasing worldwide, while most of them remain without effective treatment. Pharmacological and molecular therapy development is hampered by the lack of effective drug delivery into the posterior segment of the eye. Among molecular approaches, RNA-interference (RNAi) features strong advantages, yet delivering it to the inner layer of the retina appears extremely challenging. To address this, we developed an o...
Source
#1Merve Sen (University of Tübingen)H-Index: 2
#2Marco BassettoH-Index: 1
Last. Blanca Arango-Gonzalez (University of Tübingen)H-Index: 18
view all 6 authors...
The use of synthetic RNA for research purposes as well as RNA-based therapy and vaccination has gained increasing importance. Given the anatomical seclusion of the eye, small interfering RNA (siRNA)-induced gene silencing bears great potential for targeted reduction of pathological gene expression that may allow rational treatment of chronic eye diseases in the future. However, there is yet an unmet need for techniques providing safe and efficient siRNA delivery to the retina. We used magnetic n...
3 CitationsSource
#1Blanca Arango-Gonzalez (University of Tübingen)H-Index: 18
#2Merve Sen (University of Tübingen)H-Index: 2
Last. Marius Ueffing (University of Tübingen)H-Index: 72
view all 15 authors...
Due to continuously high production rates of rhodopsin (RHO) and high metabolic activity, photoreceptor neurons are especially vulnerable to defects in proteostasis. A proline to histidine substitution at position 23 (P23H) leads to production of structurally misfolded RHO, causing the most common form of autosomal dominant Retinitis Pigmentosa (adRP) in North America. The AAA-ATPase valosin-containing protein (VCP) extracts misfolded proteins from the ER membrane for cytosolic degradation. Here...
4 CitationsSource
#1Merve Sen (University of Tübingen)H-Index: 2
#2Oksana KutsyrH-Index: 4
Last. Marius Ueffing (University of Tübingen)H-Index: 72
view all 7 authors...
Source
#2Merve Sen (University of Tübingen)H-Index: 2
Last. Marius UeffingH-Index: 72
view all 7 authors...
#1Merve Sen (University of Tübingen)H-Index: 2
#2Oksana Kutsyr (University of Alicante)H-Index: 4
Last. Marius UeffingH-Index: 72
view all 7 authors...