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From roadmap to a sustainable end-to-end individualized therapy pathway
Anneliene H. Jonker , Elena-Alexandra Tataru , David P. Dimmock , Alison Bateman-House , Holm Graessner , Gareth Baynam , Erika F....
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
Kiran Musunuru, M.D., Ph.D. , Sarah A. Grandinette, B.S. , Xiao Wang, Ph.D. , Taylor R. Hudson, M.S. , Kevin Briseno, B.S. , Anne Marie...
The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing
Jillian Belgrad , Erin McConnell , Stef Leonard , Nicole Nolen , Marlen C Lauffer , Jonathan K Watts , Timothy Yu , Winston X Yan ,...
Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
David Cheerie , Margaret M. Meserve , Danique Beijer , Charu Kaiwar , Logan Newton , Ana Lisa Taylor Tavares , Aubrie Soucy Verran ,...
The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK
Daniel J. O’Connor , Parker Moss , Matthew Wood , Martin Murphy , Michael Parker , Nicola Blackwood , Matthew A. Brown , Deb Lancaster ,...
Personalized medicine for rare neurogenetic disorders: can we make it happen?
Agnies M van Eeghen , Hilgo Bruining , Nicole I Wolf , Arthur A Bergen , Riekelt H Houtkooper , Mieke M van Haelst , Clara D van...
A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases
Olivia Kim-McManus, Joseph G. Gleeson, Laurence Mignon, Amena Smith Fine, Winston Yan, Nicole Nolen, Scott Demarest, Elizabeth...
The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap
Anneliene H. Jonker, Elena-Alexandra Tataru, Holm Graessner, David Dimmock , Adam Jaffe, Gareth Baynam, James Davies, Shruti Mitkus,...
N-of-1 Studies in an Era of Precision Medicine
Erika Augustine, Timothy Yu, Richard Finkel ( JAMA Network ) September 25, 2024 Genetically individualized N-of-1 trials have recently...
Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics
Annemieke Aartsma-Rus, Rob W.J. Collin, Ype Elgersma, Marlen C. Lauffer, Willeke van Roon-Mom ( Therapeutic Advances in Rare Disease )...
A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides
Daniel O'Reilly, Willeke van Roon-Mom, Annemieke Aartsma-Rus; N = 1 Collaborative ( Nucleic Acid Therapeutics ) August 7, 2024...
Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing
Sara Aguti, Shuzhi Cheng, Pierpaolo Ala, Sean Briggs, Francesco Muntoni, Haiyan Zhou ( Molecular Therapy Nucleic Acids ) June 4, 2024...
How to pay for individualized genetic medicines
Julia M. Y. Pian, Nana Owusu, Julia Vitarello, Winston X. Yan, Andrew W. Lo & Timothy W. Yu (Nature Medicine) June 19, 2024 For precision...
Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments
Bianca Zardetto, Marlen C. Lauffer, Willeke van Roon-Mom, and Annemieke Aartsma-Rus on behalf of the Dutch Center for RNA Therapeutics,...
Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders
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...
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...
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....
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)...
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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