Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX

Ocular diseases create a significant economic burden and decrease in quality of life worldwide. Drugs and carrier molecules that penetrate ocular tissues after intravenous administration are needed for more efficient and patient-friendly treatment of ocular diseases. Here, ocular barrier-penetrating...

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Main Authors: Sonja Korhonen, Katja Stenberg, Umair Seemab, Piia Bartos, Katariina Mäkiniemi, Jørgen Kjems, Daniel Miotto Dupont, Astrid Subrizi
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Nucleic Acids
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Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124002397
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author Sonja Korhonen
Katja Stenberg
Umair Seemab
Piia Bartos
Katariina Mäkiniemi
Jørgen Kjems
Daniel Miotto Dupont
Astrid Subrizi
author_facet Sonja Korhonen
Katja Stenberg
Umair Seemab
Piia Bartos
Katariina Mäkiniemi
Jørgen Kjems
Daniel Miotto Dupont
Astrid Subrizi
author_sort Sonja Korhonen
collection DOAJ
description Ocular diseases create a significant economic burden and decrease in quality of life worldwide. Drugs and carrier molecules that penetrate ocular tissues after intravenous administration are needed for more efficient and patient-friendly treatment of ocular diseases. Here, ocular barrier-penetrating aptamers were selected through the utilization of in vivo SELEX and intravenous injection in rats. Three aptamers—Apt1, Apt2, and Apt5—were chosen based on their specific accumulation in vascularized ocular tissues and further characterized for their in vivo biodistribution using quantitative reverse-transcription PCR (RT-qPCR). A statistically significant difference between ΔCt values of ocular and control tissues with Apt2 (p < 0.0001) and Apt5 (p < 0.0001) was observed. Interestingly, Apt1 was the most abundant aptamer in the sequencing pool, but it did not show a statistically significant difference in in vivo biodistribution between ocular and control tissues. Overall, this study established a functional in vivo SELEX method for discovering ocular tissue targeting aptamers.
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spelling doaj-art-f5d64ecff7614091bfcc85a7b1d0e6be2025-08-20T01:48:03ZengElsevierMolecular Therapy: Nucleic Acids2162-25312024-12-0135410235210.1016/j.omtn.2024.102352Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEXSonja Korhonen0Katja Stenberg1Umair Seemab2Piia Bartos3Katariina Mäkiniemi4Jørgen Kjems5Daniel Miotto Dupont6Astrid Subrizi7School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonrinne 1 C, 70210 Kuopio, Finland; Corresponding author: Sonja Korhonen, School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonrinne 1 C, 70210 Kuopio, Finland.School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonrinne 1 C, 70210 Kuopio, FinlandSchool of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonrinne 1 C, 70210 Kuopio, FinlandSchool of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonrinne 1 C, 70210 Kuopio, FinlandSchool of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonrinne 1 C, 70210 Kuopio, FinlandInterdisciplinary Nanoscience Center (iNANO), Department of Molecular Biology and Genetics, Aarhus University, Gustav Wieds Vej 14, 8000 Aarhus C, DenmarkInterdisciplinary Nanoscience Center (iNANO), Department of Molecular Biology and Genetics, Aarhus University, Gustav Wieds Vej 14, 8000 Aarhus C, DenmarkSchool of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonrinne 1 C, 70210 Kuopio, FinlandOcular diseases create a significant economic burden and decrease in quality of life worldwide. Drugs and carrier molecules that penetrate ocular tissues after intravenous administration are needed for more efficient and patient-friendly treatment of ocular diseases. Here, ocular barrier-penetrating aptamers were selected through the utilization of in vivo SELEX and intravenous injection in rats. Three aptamers—Apt1, Apt2, and Apt5—were chosen based on their specific accumulation in vascularized ocular tissues and further characterized for their in vivo biodistribution using quantitative reverse-transcription PCR (RT-qPCR). A statistically significant difference between ΔCt values of ocular and control tissues with Apt2 (p < 0.0001) and Apt5 (p < 0.0001) was observed. Interestingly, Apt1 was the most abundant aptamer in the sequencing pool, but it did not show a statistically significant difference in in vivo biodistribution between ocular and control tissues. Overall, this study established a functional in vivo SELEX method for discovering ocular tissue targeting aptamers.http://www.sciencedirect.com/science/article/pii/S2162253124002397MT: Delivery StrategiesSELEXaptamerseyeocular targetingintravenous administration
spellingShingle Sonja Korhonen
Katja Stenberg
Umair Seemab
Piia Bartos
Katariina Mäkiniemi
Jørgen Kjems
Daniel Miotto Dupont
Astrid Subrizi
Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX
Molecular Therapy: Nucleic Acids
MT: Delivery Strategies
SELEX
aptamers
eye
ocular targeting
intravenous administration
title Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX
title_full Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX
title_fullStr Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX
title_full_unstemmed Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX
title_short Targeting ocular tissues with intravenously administered aptamers selected by in vivo SELEX
title_sort targeting ocular tissues with intravenously administered aptamers selected by in vivo selex
topic MT: Delivery Strategies
SELEX
aptamers
eye
ocular targeting
intravenous administration
url http://www.sciencedirect.com/science/article/pii/S2162253124002397
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