Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease

Serine/arginine-protein kinase 1 (SRPK1) regulates alternative splicing of VEGF-A to pro-angiogenic isoforms and SRPK1 inhibition can restore the balance of pro/antiangiogenic isoforms to normal physiological levels. The lack of potency and selectivity of available compounds has limited development...

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Main Authors: Batson, Jennifer, Toop, Hamish D., Redondo, Clara, Babaei-Jadidi, Roya, Chaikaud, Apirat, Wearmouth, Stephen F., Gibbons, Brian, Allen, Claire, Tallant, Cynthia, Zhang, Jingxue, Du, Chunyun, Hancox, Jules C., Hawtrey, Tom, Da Rocha, Joana, Griffith, Renate, Knapp, Stefan, Bates, David O., Morris, Jonathan C.
Format: Article
Published: American Chemical Society 2017
Online Access:https://eprints.nottingham.ac.uk/40843/
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author Batson, Jennifer
Toop, Hamish D.
Redondo, Clara
Babaei-Jadidi, Roya
Chaikaud, Apirat
Wearmouth, Stephen F.
Gibbons, Brian
Allen, Claire
Tallant, Cynthia
Zhang, Jingxue
Du, Chunyun
Hancox, Jules C.
Hawtrey, Tom
Da Rocha, Joana
Griffith, Renate
Knapp, Stefan
Bates, David O.
Morris, Jonathan C.
author_facet Batson, Jennifer
Toop, Hamish D.
Redondo, Clara
Babaei-Jadidi, Roya
Chaikaud, Apirat
Wearmouth, Stephen F.
Gibbons, Brian
Allen, Claire
Tallant, Cynthia
Zhang, Jingxue
Du, Chunyun
Hancox, Jules C.
Hawtrey, Tom
Da Rocha, Joana
Griffith, Renate
Knapp, Stefan
Bates, David O.
Morris, Jonathan C.
author_sort Batson, Jennifer
building Nottingham Research Data Repository
collection Online Access
description Serine/arginine-protein kinase 1 (SRPK1) regulates alternative splicing of VEGF-A to pro-angiogenic isoforms and SRPK1 inhibition can restore the balance of pro/antiangiogenic isoforms to normal physiological levels. The lack of potency and selectivity of available compounds has limited development of SRPK1 inhibitors, with the control of alternative splicing by splicing factor-specific kinases yet to be translated. We present here compounds that occupy a binding pocket created by the unique helical insert of SRPK1, and trigger a backbone flip in the hinge region, that results in potent (<10 nM) and selective inhibition of SRPK1 kinase activity. Treatment with these inhibitors inhibited SRPK1 activity and phosphorylation of serine/arginine splicing factor 1 (SRSF1), resulting in alternative splicing of VEGF-A from pro-angiogenic to antiangiogenic isoforms. This property resulted in potent inhibition of blood vessel growth in models of choroidal angiogenesis in vivo. This work identifies tool compounds for splice isoform selective targeting of pro-angiogenic VEGF, which may lead to new therapeutic strategies for a diversity of diseases where dysfunctional splicing drives disease development.
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spelling nottingham-408432024-08-15T15:21:28Z https://eprints.nottingham.ac.uk/40843/ Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease Batson, Jennifer Toop, Hamish D. Redondo, Clara Babaei-Jadidi, Roya Chaikaud, Apirat Wearmouth, Stephen F. Gibbons, Brian Allen, Claire Tallant, Cynthia Zhang, Jingxue Du, Chunyun Hancox, Jules C. Hawtrey, Tom Da Rocha, Joana Griffith, Renate Knapp, Stefan Bates, David O. Morris, Jonathan C. Serine/arginine-protein kinase 1 (SRPK1) regulates alternative splicing of VEGF-A to pro-angiogenic isoforms and SRPK1 inhibition can restore the balance of pro/antiangiogenic isoforms to normal physiological levels. The lack of potency and selectivity of available compounds has limited development of SRPK1 inhibitors, with the control of alternative splicing by splicing factor-specific kinases yet to be translated. We present here compounds that occupy a binding pocket created by the unique helical insert of SRPK1, and trigger a backbone flip in the hinge region, that results in potent (<10 nM) and selective inhibition of SRPK1 kinase activity. Treatment with these inhibitors inhibited SRPK1 activity and phosphorylation of serine/arginine splicing factor 1 (SRSF1), resulting in alternative splicing of VEGF-A from pro-angiogenic to antiangiogenic isoforms. This property resulted in potent inhibition of blood vessel growth in models of choroidal angiogenesis in vivo. This work identifies tool compounds for splice isoform selective targeting of pro-angiogenic VEGF, which may lead to new therapeutic strategies for a diversity of diseases where dysfunctional splicing drives disease development. American Chemical Society 2017-01-30 Article PeerReviewed Batson, Jennifer, Toop, Hamish D., Redondo, Clara, Babaei-Jadidi, Roya, Chaikaud, Apirat, Wearmouth, Stephen F., Gibbons, Brian, Allen, Claire, Tallant, Cynthia, Zhang, Jingxue, Du, Chunyun, Hancox, Jules C., Hawtrey, Tom, Da Rocha, Joana, Griffith, Renate, Knapp, Stefan, Bates, David O. and Morris, Jonathan C. (2017) Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease. ACS Chemical Biology, 12 (3). pp. 825-832. ISSN 1554-8937 http://pubs.acs.org/doi/abs/10.1021/acschembio.6b01048 doi:10.1021/acschembio.6b01048 doi:10.1021/acschembio.6b01048
spellingShingle Batson, Jennifer
Toop, Hamish D.
Redondo, Clara
Babaei-Jadidi, Roya
Chaikaud, Apirat
Wearmouth, Stephen F.
Gibbons, Brian
Allen, Claire
Tallant, Cynthia
Zhang, Jingxue
Du, Chunyun
Hancox, Jules C.
Hawtrey, Tom
Da Rocha, Joana
Griffith, Renate
Knapp, Stefan
Bates, David O.
Morris, Jonathan C.
Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease
title Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease
title_full Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease
title_fullStr Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease
title_full_unstemmed Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease
title_short Development of potent, selective SRPK1 inhibitors as potential topical therapeutics for neovascular eye disease
title_sort development of potent, selective srpk1 inhibitors as potential topical therapeutics for neovascular eye disease
url https://eprints.nottingham.ac.uk/40843/
https://eprints.nottingham.ac.uk/40843/
https://eprints.nottingham.ac.uk/40843/