Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders
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Marlen C. Lauffer, Willeke van Roon-Mom, Annemieke Aartsma-Rus & N=1 Collaborative (Communications Medicine) January 5, 2024 Abstract...
A Multistakeholder Perspective on Advancing Individualized Therapeutics
Michael Pacanowski, Julia Vitarello, Insoo Hyun, Timothy Yu, Issam Zineh August 24, 2023 Precision medicine has evolved from the...
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A framework for individualized splice-switching oligonucleotide therapy
Jinkuk Kim, Sijae Woo, Claudio M. de Gusmao, Boxun Zhao, Diana H. Chin, Renata L. DiDonato, Minh A. Nguyen, Tojo Nakayama, Chunguang...
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Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1
Annemieke Aartsma-Rus, Alejandro Garanto, Willeke van Roon-Mom, Erin M. McConnell, Victoria Suslovitch, Winston X. Yan, Jonathan K....
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Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic later
Vladislav A. Korobeynikov, Alexander K. Lyashchenko, Beatriz Blanco-Redondo, Paymaan Jafar-Nejad & Neil A. Shneider (Nature Medicine)...
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New antisense oligonucleotide therapies reach first base in ALS
Elia R. Lopez, William F. Borschel & Bryan J. Traynor January 24, 2022 Two studies highlight the evolution of antisense oligonucleotide...
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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
Hélène Tran, Michael P. Moazami, Huiya Yang, Diane McKenna-Yasek, Catherine L. Douthwright, Courtney Pinto, Jake Metterville, Minwook...
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OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
This briefing document aims to outline the current state of the art of oligonucleotide therapies for those planning to develop...
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Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Jinkuk Kim, Ph.D., Chunguang Hu, M.D., Ph.D., Christelle Moufawad El Achkar, M.D., Lauren E. Black, Ph.D., Julie Douville, Ph.D., Austin...
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Drug Regulation in the Era of Individualized Therapies
Janet Woodcock, M.D., and Peter Marks, M.D., Ph.D. (NEJM) October 24, 2019 Kim et al., in a report now published in the Journal,1 ...
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