Nucleic acid therapeutics

Keyphrases

Publications

volume 34, number 1, 2024

Martin D Requena, Amy Yan, Telmo Llanga, Bruce A Sullenger
Reversible Aptamer Staining, Sorting, and Cleaning of Cells (Clean FACS) with Antidote Oligonucleotide or Nuclease Yields Fully Responsive Cells. Nucleic acid therapeutics 34 (1) (2024)
Li Li
"Goldilocks Modifications" for mRNA Therapeutics Won the Nobel Prize. Nucleic acid therapeutics 34 (1) (2024)
Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordoʹ, Annemieke M Aartsma-Rus
Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic acid therapeutics 34 (1) (2024)
Tom Metz, Fijs W B van Leeuwen, Ernst Suidgeest, Esmée Nieuwenhuize, Thomas de Vlaam, Daniel Curtis, Tsinatkeab T Hailu, Louise van der Weerd, Willeke M C van Roon-Mom
Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice. Nucleic acid therapeutics 34 (1) (2024)
Brenda F Baker, Shuting Xia, Wesley Partridge, Jeffery A Engelhardt, Sotirios Tsimikas, Stanley T Crooke, Sanjay Bhanot, Richard S Geary
Safety and Tolerability of GalNAc 3 -Conjugated Antisense Drugs Compared to the Same-Sequence 2'- O -Methoxyethyl-Modified Antisense Drugs: Results from an Integrated Assessment of Phase 1 Clinical Trial Data. Nucleic acid therapeutics 34 (1) (2024)
Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordoʹ, Annemieke M Aartsma-Rus
Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic acid therapeutics 34 (1) (2024)
Li Li
"Goldilocks Modifications" for mRNA Therapeutics Won the Nobel Prize. Nucleic acid therapeutics 34 (1) (2024)
Li Li
"Goldilocks Modifications" for mRNA Therapeutics Won the Nobel Prize. Nucleic acid therapeutics 34 (1) (2024)
Martin D Requena, Amy Yan, Telmo Llanga, Bruce A Sullenger
Reversible Aptamer Staining, Sorting, and Cleaning of Cells (Clean FACS) with Antidote Oligonucleotide or Nuclease Yields Fully Responsive Cells. Nucleic acid therapeutics 34 (1) (2024)

volume 34, number 2, 2024

Natalia V Klementieva, Evgenii A Lunev, Anna A Shmidt, Elizaveta M Loseva, Irina M Savchenko, Ekaterina A Svetlova, Ivan I Galkin, Anna V Polikarpova, Evgeny V Usachev, Svetlana G Vassilieva, Valeria I Marina, Marina A Dzhenkova, Anna D Romanova, Anton V Agutin, Anna A Timakova, Denis A Reshetov, Tatiana V Egorova, Maryana V Bardina
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro . Nucleic acid therapeutics 34 (2) (2024)
Sudhir Agrawal
Considerations for Creating the Next Generation of RNA Therapeutics: Oligonucleotide Chemistry and Innate Immune Responses to Nucleic Acids. Nucleic acid therapeutics 34 (2) (2024)
Nuria Suárez-Herrera, Alejandro Garanto, Rob W J Collin
Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease. Nucleic acid therapeutics 34 (2) (2024)
Natalia V Klementieva, Evgenii A Lunev, Anna A Shmidt, Elizaveta M Loseva, Irina M Savchenko, Ekaterina A Svetlova, Ivan I Galkin, Anna V Polikarpova, Evgeny V Usachev, Svetlana G Vassilieva, Valeria I Marina, Marina A Dzhenkova, Anna D Romanova, Anton V Agutin, Anna A Timakova, Denis A Reshetov, Tatiana V Egorova, Maryana V Bardina
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro . Nucleic acid therapeutics 34 (2) (2024)
Sudhir Agrawal
Considerations for Creating the Next Generation of RNA Therapeutics: Oligonucleotide Chemistry and Innate Immune Responses to Nucleic Acids. Nucleic acid therapeutics 34 (2) (2024)
Nuria Suárez-Herrera, Alejandro Garanto, Rob W J Collin
Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease. Nucleic acid therapeutics 34 (2) (2024)
Xuan Zhou, Xianglin Shi, Yannick Fillon, Firoz Antia, Thomas Pickel, Jing Yang, William Zhang, Armin Delavari, Jiabao Zhang
Simplified Oligonucleotide Phosphorus Deprotection Process with Reduced 3-(2-Cyanoethyl) Thymidine Impurities. Nucleic acid therapeutics 34 (2) (2024)
Jillian Belgrad, Hassan H Fakih, Anastasia Khvorova
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation. Nucleic acid therapeutics 34 (2) (2024)
Jillian Belgrad, Hassan H Fakih, Anastasia Khvorova
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation. Nucleic acid therapeutics 34 (2) (2024)
Nuria Suárez-Herrera, Alejandro Garanto, Rob W J Collin
Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease. Nucleic acid therapeutics 34 (2) (2024)
Natalia V Klementieva, Evgenii A Lunev, Anna A Shmidt, Elizaveta M Loseva, Irina M Savchenko, Ekaterina A Svetlova, Ivan I Galkin, Anna V Polikarpova, Evgeny V Usachev, Svetlana G Vassilieva, Valeria I Marina, Marina A Dzhenkova, Anna D Romanova, Anton V Agutin, Anna A Timakova, Denis A Reshetov, Tatiana V Egorova, Maryana V Bardina
RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro . Nucleic acid therapeutics 34 (2) (2024)
Jillian Belgrad, Hassan H Fakih, Anastasia Khvorova
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation. Nucleic acid therapeutics 34 (2) (2024)
Nuria Suárez-Herrera, Alejandro Garanto, Rob W J Collin
Understanding and Rescuing the Splicing Defect Caused by the Frequent ABCA4 Variant c.4253+43G>A Underlying Stargardt Disease. Nucleic acid therapeutics 34 (2) (2024)
Jillian Belgrad, Hassan H Fakih, Anastasia Khvorova
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation. Nucleic acid therapeutics 34 (2) (2024)
Jillian Belgrad, Hassan H Fakih, Anastasia Khvorova
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation. Nucleic acid therapeutics 34 (2) (2024)
Xuan Zhou, Xianglin Shi, Yannick Fillon, Firoz Antia, Thomas Pickel, Jing Yang, William Zhang, Armin Delavari, Jiabao Zhang
Simplified Oligonucleotide Phosphorus Deprotection Process with Reduced 3-(2-Cyanoethyl) Thymidine Impurities. Nucleic acid therapeutics 34 (2) (2024)
Xuan Zhou, Xianglin Shi, Yannick Fillon, Firoz Antia, Thomas Pickel, Jing Yang, William Zhang, Armin Delavari, Jiabao Zhang
Simplified Oligonucleotide Phosphorus Deprotection Process with Reduced 3-(2-Cyanoethyl) Thymidine Impurities. Nucleic acid therapeutics 34 (2) (2024)

volume 34, number 3, 2024

Laura V Croft, Mark Fisher, Tabassum Khair Barbhuiya, Serene El-Kamand, Samuel Beard, Aleksandra Rajapakse, Roland Gamsjaeger, Liza Cubeddu, Emma Bolderson, Kenneth J O'Byrne, Derek Richard, Neha S Gandhi
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2'- O -Methyl Modified Single-Stranded Oligonucleotides. Nucleic acid therapeutics 34 (3) (2024)
Brian J Thomas, Caitlyn Guldenpfennig, Mark A Daniels, Donald H Burke, David Porciani
Multiplexed In Vivo Screening Using Barcoded Aptamer Technology to Identify Oligonucleotide-Based Targeting Reagents. Nucleic acid therapeutics 34 (3) (2024)
Adi Idris, Surya Shrivastava, Aroon Supramaniam, Roslyn M Ray, Galina Shevchenko, Dhruba Acharya, Nigel A J McMillan, Kevin V Morris
Extracellular Vesicles Loaded with Long Antisense RNAs Repress Severe Acute Respiratory Syndrome Coronavirus 2 Infection. Nucleic acid therapeutics 34 (3) (2024)
Zelia Corradi, Rebekkah J Hitti-Malin, Laura A de Rooij, Alejandro Garanto, Rob W J Collin, Frans P M Cremers
Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in ABCA4 . Nucleic acid therapeutics 34 (3) (2024)
Laura V Croft, Mark Fisher, Tabassum Khair Barbhuiya, Serene El-Kamand, Samuel Beard, Aleksandra Rajapakse, Roland Gamsjaeger, Liza Cubeddu, Emma Bolderson, Kenneth J O'Byrne, Derek Richard, Neha S Gandhi
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2'- O -Methyl Modified Single-Stranded Oligonucleotides. Nucleic acid therapeutics 34 (3) (2024)
Brian J Thomas, Caitlyn Guldenpfennig, Mark A Daniels, Donald H Burke, David Porciani
Multiplexed In Vivo Screening Using Barcoded Aptamer Technology to Identify Oligonucleotide-Based Targeting Reagents. Nucleic acid therapeutics 34 (3) (2024)
Adi Idris, Surya Shrivastava, Aroon Supramaniam, Roslyn M Ray, Galina Shevchenko, Dhruba Acharya, Nigel A J McMillan, Kevin V Morris
Extracellular Vesicles Loaded with Long Antisense RNAs Repress Severe Acute Respiratory Syndrome Coronavirus 2 Infection. Nucleic acid therapeutics 34 (3) (2024)
Ainhoa Martínez-Pizarro, Mar Álvarez, Maja Dembic, Caroline A Lindegaard, Margarita Castro, Eva Richard, Brage S Andresen, Lourdes Ruiz Desviat
Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria. Nucleic acid therapeutics 34 (3) (2024)
Brian J Thomas, Caitlyn Guldenpfennig, Mark A Daniels, Donald H Burke, David Porciani
Multiplexed In Vivo Screening Using Barcoded Aptamer Technology to Identify Oligonucleotide-Based Targeting Reagents. Nucleic acid therapeutics 34 (3) (2024)
Zelia Corradi, Rebekkah J Hitti-Malin, Laura A de Rooij, Alejandro Garanto, Rob W J Collin, Frans P M Cremers
Antisense Oligonucleotide-Based Rescue of Complex Intronic Splicing Defects in ABCA4 . Nucleic acid therapeutics 34 (3) (2024)
Brian J Thomas, Caitlyn Guldenpfennig, Mark A Daniels, Donald H Burke, David Porciani
Multiplexed In Vivo Screening Using Barcoded Aptamer Technology to Identify Oligonucleotide-Based Targeting Reagents. Nucleic acid therapeutics 34 (3) (2024)
Adi Idris, Surya Shrivastava, Aroon Supramaniam, Roslyn M Ray, Galina Shevchenko, Dhruba Acharya, Nigel A J McMillan, Kevin V Morris
Extracellular Vesicles Loaded with Long Antisense RNAs Repress Severe Acute Respiratory Syndrome Coronavirus 2 Infection. Nucleic acid therapeutics 34 (3) (2024)
Ainhoa Martínez-Pizarro, Mar Álvarez, Maja Dembic, Caroline A Lindegaard, Margarita Castro, Eva Richard, Brage S Andresen, Lourdes Ruiz Desviat
Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria. Nucleic acid therapeutics 34 (3) (2024)
Adi Idris, Surya Shrivastava, Aroon Supramaniam, Roslyn M Ray, Galina Shevchenko, Dhruba Acharya, Nigel A J McMillan, Kevin V Morris
Extracellular Vesicles Loaded with Long Antisense RNAs Repress Severe Acute Respiratory Syndrome Coronavirus 2 Infection. Nucleic acid therapeutics 34 (3) (2024)

volume 34, number 4, 2024

Cy A Stein
Phosphorothioates and Me: A Lecture About My 35 Years in Oligo-World on My Receipt of the 2023 Lifetime Achievement Award of the Oligonucleotide Therapeutics Society. Nucleic acid therapeutics 34 (4) (2024)
Sarah Allen, Daniel O'Reilly, Rachael Miller, Ellen Sapp, Ashley Summers, Joseph Paquette, Dimas Echeverria Moreno, Brianna Bramato, Nicholas McHugh, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo . Nucleic acid therapeutics 34 (4) (2024)
Rui Xu, Emmanuel Njumbe Ediage, Tom Verhaeghe, Jan Snoeys, Lieve Dillen
Therapeutic siRNA Loaded to RISC as Single and Double Strands Requires an Appropriate Quantitative Assay for RISC PK Assessment. Nucleic acid therapeutics 34 (4) (2024)
Ledong Wan, Alexander J Kral, Dillon Voss, Balázs Schäfer, Kavitha Sudheendran, Mathias Danielsen, Marvin H Caruthers, Adrian R Krainer
Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids. Nucleic acid therapeutics 34 (4) (2024)
Sarah Allen, Daniel O'Reilly, Rachael Miller, Ellen Sapp, Ashley Summers, Joseph Paquette, Dimas Echeverria Moreno, Brianna Bramato, Nicholas McHugh, Ken Yamada, Neil Aronin, Marian DiFiglia, Anastasia Khvorova
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo . Nucleic acid therapeutics 34 (4) (2024)
Anna Lisa Scardovi, Damiano Bartolucci, Luca Montemurro, Sonia Bortolotti, Silvia Angelucci, Camilla Amadesi, Giammario Nieddu, Sean Oosterholt, Lucia Cerisoli, Oscar Della Pasqua, Patrizia Hrelia, Roberto Tonelli
Preclinical Pharmacokinetics in Tumors and Normal Tissues of the Antigene PNA Oligonucleotide MYCN-Inhibitor BGA002. Nucleic acid therapeutics 34 (4) (2024)
Ledong Wan, Alexander J Kral, Dillon Voss, Balázs Schäfer, Kavitha Sudheendran, Mathias Danielsen, Marvin H Caruthers, Adrian R Krainer
Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids. Nucleic acid therapeutics 34 (4) (2024)
Cy A Stein
Phosphorothioates and Me: A Lecture About My 35 Years in Oligo-World on My Receipt of the 2023 Lifetime Achievement Award of the Oligonucleotide Therapeutics Society. Nucleic acid therapeutics 34 (4) (2024)
Anna Lisa Scardovi, Damiano Bartolucci, Luca Montemurro, Sonia Bortolotti, Silvia Angelucci, Camilla Amadesi, Giammario Nieddu, Sean Oosterholt, Lucia Cerisoli, Oscar Della Pasqua, Patrizia Hrelia, Roberto Tonelli
Preclinical Pharmacokinetics in Tumors and Normal Tissues of the Antigene PNA Oligonucleotide MYCN-Inhibitor BGA002. Nucleic acid therapeutics 34 (4) (2024)
Rui Xu, Emmanuel Njumbe Ediage, Tom Verhaeghe, Jan Snoeys, Lieve Dillen
Therapeutic siRNA Loaded to RISC as Single and Double Strands Requires an Appropriate Quantitative Assay for RISC PK Assessment. Nucleic acid therapeutics 34 (4) (2024)