QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa: a preclinical study
Dystrophic epidermolysis bullosa (DEB) is a blistering skin disease caused by mutations in the gene COL7A1 encoding collagen VII. DEB can be inherited as recessive DEB (RDEB) or dominant DEB (DDEB) and is associated with a high wound burden. Perpetual cycles of wounding and healing drive fibrosis in...
Saved in:
| Main Authors: | , , , , , , , , , , , , , , , , , , |
|---|---|
| Format: | Article (Journal) |
| Language: | English |
| Published: |
2021
|
| In: |
The journal of investigative dermatology
Year: 2020, Volume: 141, Issue: 4, Pages: 883-893 |
| ISSN: | 1523-1747 |
| DOI: | 10.1016/j.jid.2020.08.018 |
| Online Access: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1016/j.jid.2020.08.018 Verlag, lizenzpflichtig, Volltext: https://www.sciencedirect.com/science/article/pii/S0022202X20320613 
|
| Author Notes: | Olivier Bornert, Marieke Hogervorst, Pauline Nauroy, Johannes Bischof, Jim Swildens, Ioannis Athanasiou, Sara F. Tufa, Douglas R. Keene, Dimitra Kiritsi, Stefan Hainzl, Eva M. Murauer, M. Peter Marinkovich, Gerard Platenburg, Ingrid Hausser, Verena Wally, Tita Ritsema, Ulrich Koller, Elisabeth M. Haisma and Alexander Nyström |
MARC
| LEADER | 00000caa a2200000 c 4500 | ||
|---|---|---|---|
| 001 | 1757545247 | ||
| 003 | DE-627 | ||
| 005 | 20241230014016.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 210510r20212020xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1016/j.jid.2020.08.018 |2 doi | |
| 035 | |a (DE-627)1757545247 | ||
| 035 | |a (DE-599)KXP1757545247 | ||
| 035 | |a (OCoLC)1341408648 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 084 | |a 33 |2 sdnb | ||
| 100 | 1 | |a Bornert, Olivier |e VerfasserIn |0 (DE-588)1233320513 |0 (DE-627)1757654941 |4 aut | |
| 245 | 1 | 0 | |a QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa |b a preclinical study |c Olivier Bornert, Marieke Hogervorst, Pauline Nauroy, Johannes Bischof, Jim Swildens, Ioannis Athanasiou, Sara F. Tufa, Douglas R. Keene, Dimitra Kiritsi, Stefan Hainzl, Eva M. Murauer, M. Peter Marinkovich, Gerard Platenburg, Ingrid Hausser, Verena Wally, Tita Ritsema, Ulrich Koller, Elisabeth M. Haisma and Alexander Nyström |
| 246 | 3 | 0 | |a three hundred thirteen |
| 264 | 1 | |c 2021 | |
| 300 | |a 11 | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Das PDF enthält zusätzlich einen Anhang von 6 Seiten (893.e1-893.e6) | ||
| 500 | |a Corrected proof published online 17 November 2020 | ||
| 500 | |a Gesehen am 10.05.2021 | ||
| 520 | |a Dystrophic epidermolysis bullosa (DEB) is a blistering skin disease caused by mutations in the gene COL7A1 encoding collagen VII. DEB can be inherited as recessive DEB (RDEB) or dominant DEB (DDEB) and is associated with a high wound burden. Perpetual cycles of wounding and healing drive fibrosis in DDEB and RDEB, as well as the formation of a tumor-permissive microenvironment. Prolonging wound-free episodes by improving the quality of wound healing would therefore confer substantial benefit for individuals with DEB. The collagenous domain of collagen VII is encoded by 82 in-frame exons, which makes splice-modulation therapies attractive for DEB. Indeed, antisense oligonucleotide-based exon skipping has shown promise for RDEB. However, the suitability of antisense oligonucleotides for treatment of DDEB remains unexplored. Here, we developed QR-313, a clinically applicable, potent antisense oligonucleotide specifically targeting exon 73. We show the feasibility of topical delivery of QR-313 in a carbomer-composed gel for treatment of wounds to restore collagen VII abundance in human RDEB skin. Our data reveal that QR-313 also shows direct benefit for DDEB caused by exon 73 mutations. Thus, the same topically applied therapeutic could be used to improve the wound healing quality in RDEB and DDEB. | ||
| 534 | |c 2020 | ||
| 700 | 1 | |a Hogervorst, Marieke |e VerfasserIn |4 aut | |
| 700 | 1 | |8 1\p |a Nauroy, Pauline |e VerfasserIn |0 (DE-588)1198905727 |0 (DE-627)1681098687 |4 aut | |
| 700 | 1 | |8 2\p |a Bischof, Johannes |d 1963- |e VerfasserIn |0 (DE-588)128914807 |0 (DE-627)385330863 |0 (DE-576)297397427 |4 aut | |
| 700 | 1 | |a Swildens, Jim |e VerfasserIn |4 aut | |
| 700 | 1 | |8 3\p |a Athanasiou, Ioannis |e VerfasserIn |0 (DE-588)1176721380 |0 (DE-627)1047750538 |0 (DE-576)516774298 |4 aut | |
| 700 | 1 | |a Tufa, Sara F. |e VerfasserIn |4 aut | |
| 700 | 1 | |a Keene, Douglas R. |e VerfasserIn |4 aut | |
| 700 | 1 | |8 4\p |a Kiritsi, Dimitra |d 1983- |e VerfasserIn |0 (DE-588)1015004806 |0 (DE-627)668336684 |0 (DE-576)349965064 |4 aut | |
| 700 | 1 | |a Hainzl, Stefan |e VerfasserIn |4 aut | |
| 700 | 1 | |a Murauer, Eva M. |e VerfasserIn |4 aut | |
| 700 | 1 | |a Marinkovich, M. Peter |e VerfasserIn |4 aut | |
| 700 | 1 | |a Platenburg, Gerard |e VerfasserIn |4 aut | |
| 700 | 1 | |a Haußer-Siller, Ingrid |d 1957- |e VerfasserIn |0 (DE-588)1058096710 |0 (DE-627)796384703 |0 (DE-576)163782377 |4 aut | |
| 700 | 1 | |a Wally, Verena |e VerfasserIn |4 aut | |
| 700 | 1 | |a Ritsema, Tita |e VerfasserIn |4 aut | |
| 700 | 1 | |a Koller, Ulrich |e VerfasserIn |4 aut | |
| 700 | 1 | |a Haisma, Elisabeth M. |e VerfasserIn |4 aut | |
| 700 | 1 | |8 5\p |a Nyström, Alexander |e VerfasserIn |0 (DE-588)1133092608 |0 (DE-627)888232934 |0 (DE-576)489106668 |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t The journal of investigative dermatology |d Amsterdam : Elsevier, 1938 |g 141(2021), 4, Seite 883-893 |h Online-Ressource |w (DE-627)320457990 |w (DE-600)2006902-9 |w (DE-576)09114289X |x 1523-1747 |7 nnas |a QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa a preclinical study |
| 773 | 1 | 8 | |g volume:141 |g year:2021 |g number:4 |g pages:883-893 |g extent:11 |a QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa a preclinical study |
| 856 | 4 | 0 | |u https://doi.org/10.1016/j.jid.2020.08.018 |x Verlag |x Resolving-System |z lizenzpflichtig |3 Volltext |
| 856 | 4 | 0 | |u https://www.sciencedirect.com/science/article/pii/S0022202X20320613 |x Verlag |z lizenzpflichtig |3 Volltext |
| 883 | |8 1\p |a cgwrk |d 20241001 |q DE-101 |u https://d-nb.info/provenance/plan#cgwrk | ||
| 883 | |8 2\p |a cgwrk |d 20241001 |q DE-101 |u https://d-nb.info/provenance/plan#cgwrk | ||
| 883 | |8 3\p |a cgwrk |d 20241001 |q DE-101 |u https://d-nb.info/provenance/plan#cgwrk | ||
| 883 | |8 4\p |a cgwrk |d 20241001 |q DE-101 |u https://d-nb.info/provenance/plan#cgwrk | ||
| 883 | |8 5\p |a cgwrk |d 20241001 |q DE-101 |u https://d-nb.info/provenance/plan#cgwrk | ||
| 951 | |a AR | ||
| 992 | |a 20210510 | ||
| 993 | |a Article | ||
| 994 | |a 2021 | ||
| 998 | |g 1058096710 |a Haußer-Siller, Ingrid |m 1058096710:Haußer-Siller, Ingrid |d 910000 |d 912000 |e 910000PH1058096710 |e 912000PH1058096710 |k 0/910000/ |k 1/910000/912000/ |p 14 | ||
| 999 | |a KXP-PPN1757545247 |e 3927126632 | ||
| BIB | |a Y | ||
| SER | |a journal | ||
| JSO | |a {"id":{"doi":["10.1016/j.jid.2020.08.018"],"eki":["1757545247"]},"note":["Das PDF enthält zusätzlich einen Anhang von 6 Seiten (893.e1-893.e6)","Corrected proof published online 17 November 2020","Gesehen am 10.05.2021"],"recId":"1757545247","language":["eng"],"name":{"displayForm":["Olivier Bornert, Marieke Hogervorst, Pauline Nauroy, Johannes Bischof, Jim Swildens, Ioannis Athanasiou, Sara F. Tufa, Douglas R. Keene, Dimitra Kiritsi, Stefan Hainzl, Eva M. Murauer, M. Peter Marinkovich, Gerard Platenburg, Ingrid Hausser, Verena Wally, Tita Ritsema, Ulrich Koller, Elisabeth M. Haisma and Alexander Nyström"]},"relHost":[{"type":{"media":"Online-Ressource","bibl":"periodical"},"disp":"QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa a preclinical studyThe journal of investigative dermatology","physDesc":[{"extent":"Online-Ressource"}],"note":["Gesehen am 16.02.16"],"id":{"issn":["1523-1747"],"eki":["320457990"],"zdb":["2006902-9"]},"recId":"320457990","corporate":[{"display":"Society for Investigative Dermatology","role":"isb","roleDisplay":"Herausgebendes Organ"}],"origin":[{"publisherPlace":"Amsterdam ; Oxford [u.a.] ; [Erscheinungsort nicht ermittelbar] ; Basingstoke [u.a.]","dateIssuedKey":"1938","dateIssuedDisp":"1938-","publisher":"Elsevier ; Blackwell ; Wiley-Blackwell ; Nature Publishing Group"}],"title":[{"title":"The journal of investigative dermatology","title_sort":"journal of investigative dermatology"}],"titleAlt":[{"title":"an international journal for research in cutaneous biology"}],"pubHistory":["1.1938 -"],"part":{"issue":"4","pages":"883-893","text":"141(2021), 4, Seite 883-893","volume":"141","year":"2021","extent":"11"},"name":{"displayForm":["Society for Investigative Dermatology ; European Society for Dermatological Research"]},"language":["eng"]}],"origin":[{"dateIssuedKey":"2021","dateIssuedDisp":"2021"}],"type":{"media":"Online-Ressource","bibl":"article-journal"},"title":[{"title":"QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa","title_sort":"QR-313, an antisense oligonucleotide, shows therapeutic efficacy for treatment of dominant and recessive dystrophic epidermolysis bullosa","subtitle":"a preclinical study"}],"person":[{"role":"aut","roleDisplay":"VerfasserIn","given":"Olivier","family":"Bornert","display":"Bornert, Olivier"},{"family":"Hogervorst","display":"Hogervorst, Marieke","roleDisplay":"VerfasserIn","given":"Marieke","role":"aut"},{"role":"aut","given":"Pauline","roleDisplay":"VerfasserIn","display":"Nauroy, Pauline","family":"Nauroy"},{"role":"aut","roleDisplay":"VerfasserIn","given":"Johannes","display":"Bischof, Johannes","family":"Bischof"},{"roleDisplay":"VerfasserIn","given":"Jim","role":"aut","display":"Swildens, Jim","family":"Swildens"},{"family":"Athanasiou","display":"Athanasiou, Ioannis","role":"aut","given":"Ioannis","roleDisplay":"VerfasserIn"},{"display":"Tufa, Sara F.","family":"Tufa","role":"aut","roleDisplay":"VerfasserIn","given":"Sara F."},{"family":"Keene","display":"Keene, Douglas R.","role":"aut","given":"Douglas R.","roleDisplay":"VerfasserIn"},{"roleDisplay":"VerfasserIn","given":"Dimitra","role":"aut","display":"Kiritsi, Dimitra","family":"Kiritsi"},{"role":"aut","roleDisplay":"VerfasserIn","given":"Stefan","display":"Hainzl, Stefan","family":"Hainzl"},{"role":"aut","given":"Eva M.","roleDisplay":"VerfasserIn","display":"Murauer, Eva M.","family":"Murauer"},{"role":"aut","given":"M. Peter","roleDisplay":"VerfasserIn","display":"Marinkovich, M. Peter","family":"Marinkovich"},{"roleDisplay":"VerfasserIn","given":"Gerard","role":"aut","family":"Platenburg","display":"Platenburg, Gerard"},{"display":"Haußer-Siller, Ingrid","family":"Haußer-Siller","role":"aut","given":"Ingrid","roleDisplay":"VerfasserIn"},{"display":"Wally, Verena","family":"Wally","role":"aut","roleDisplay":"VerfasserIn","given":"Verena"},{"role":"aut","roleDisplay":"VerfasserIn","given":"Tita","display":"Ritsema, Tita","family":"Ritsema"},{"role":"aut","given":"Ulrich","roleDisplay":"VerfasserIn","family":"Koller","display":"Koller, Ulrich"},{"family":"Haisma","display":"Haisma, Elisabeth M.","role":"aut","roleDisplay":"VerfasserIn","given":"Elisabeth M."},{"roleDisplay":"VerfasserIn","given":"Alexander","role":"aut","family":"Nyström","display":"Nyström, Alexander"}],"physDesc":[{"extent":"11 S."}]} | ||
| SRT | |a BORNERTOLIQR313ANANT2021 | ||