top of page
![](https://static.wixstatic.com/media/2be1ce_54be3475a7fe44828c7fd17f6650ffdd~mv2.jpeg/v1/fill/w_741,h_3000,al_c,q_90,enc_avif,quality_auto/2be1ce_54be3475a7fe44828c7fd17f6650ffdd~mv2.jpeg)
Search
![](https://static.wixstatic.com/media/c9b1c6_f138b51041c345af8cb82dd78fe22d92~mv2.jpg/v1/fill/w_250,h_248,al_c,q_30,blur_30,enc_auto/c9b1c6_f138b51041c345af8cb82dd78fe22d92~mv2.webp)
![Personalized medicine for rare neurogenetic disorders: can we make it happen?](https://static.wixstatic.com/media/c9b1c6_f138b51041c345af8cb82dd78fe22d92~mv2.jpg/v1/fill/w_305,h_303,fp_0.50_0.50,q_90,enc_auto/c9b1c6_f138b51041c345af8cb82dd78fe22d92~mv2.webp)
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...
46 views
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...
96 views
![The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap](https://static.wixstatic.com/media/c9b1c6_cf3b4346bf114bc8abd3e4da8cf90ad8~mv2.png/v1/fill/w_305,h_155,fp_0.50_0.50,q_95,enc_auto/c9b1c6_cf3b4346bf114bc8abd3e4da8cf90ad8~mv2.webp)
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,...
110 views
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...
75 views
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 )...
25 views
![A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides](https://static.wixstatic.com/media/c9b1c6_c11acee0acf04e818af25b687947403e~mv2.png/v1/fill/w_305,h_264,fp_0.50_0.50,q_95,enc_auto/c9b1c6_c11acee0acf04e818af25b687947403e~mv2.webp)
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...
36 views
![](https://static.wixstatic.com/media/c9b1c6_113abf7497f34b76a16e90142bf10c9b~mv2.jpg/v1/fill/w_250,h_250,al_c,q_30,blur_30,enc_auto/c9b1c6_113abf7497f34b76a16e90142bf10c9b~mv2.webp)
![Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing](https://static.wixstatic.com/media/c9b1c6_113abf7497f34b76a16e90142bf10c9b~mv2.jpg/v1/fill/w_306,h_306,fp_0.50_0.50,q_90,enc_auto/c9b1c6_113abf7497f34b76a16e90142bf10c9b~mv2.webp)
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...
42 views
![How to pay for individualized genetic medicines](https://static.wixstatic.com/media/c9b1c6_c22e8f77966f42b081624425e546afb5~mv2.png/v1/fill/w_305,h_200,fp_0.50_0.50,q_95,enc_auto/c9b1c6_c22e8f77966f42b081624425e546afb5~mv2.webp)
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...
80 views
![](https://static.wixstatic.com/media/c9b1c6_a57bd9635e0e49599a22b3e7b21ea166~mv2.jpg/v1/fill/w_167,h_250,al_c,q_30,blur_30,enc_auto/c9b1c6_a57bd9635e0e49599a22b3e7b21ea166~mv2.webp)
![Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments](https://static.wixstatic.com/media/c9b1c6_a57bd9635e0e49599a22b3e7b21ea166~mv2.jpg/v1/fill/w_305,h_456,fp_0.50_0.50,q_90,enc_auto/c9b1c6_a57bd9635e0e49599a22b3e7b21ea166~mv2.webp)
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,...
85 views
![](https://static.wixstatic.com/media/c9b1c6_ce871a66744f46e78f0d20913501e995~mv2.webp/v1/fill/w_224,h_250,al_c,q_30,blur_30,enc_auto/c9b1c6_ce871a66744f46e78f0d20913501e995~mv2.webp)
![Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders](https://static.wixstatic.com/media/c9b1c6_ce871a66744f46e78f0d20913501e995~mv2.webp/v1/fill/w_306,h_341,al_c,q_90,enc_auto/c9b1c6_ce871a66744f46e78f0d20913501e995~mv2.webp)
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...
90 views
![](https://static.wixstatic.com/media/c9b1c6_f6058031c75b421091b9ab862fc50619~mv2.webp/v1/fill/w_250,h_116,al_c,q_30,blur_30,enc_auto/c9b1c6_f6058031c75b421091b9ab862fc50619~mv2.webp)
![A Multistakeholder Perspective on Advancing Individualized Therapeutics](https://static.wixstatic.com/media/c9b1c6_f6058031c75b421091b9ab862fc50619~mv2.webp/v1/fill/w_305,h_142,al_c,q_90,enc_auto/c9b1c6_f6058031c75b421091b9ab862fc50619~mv2.webp)
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...
81 views
![](https://static.wixstatic.com/media/c9b1c6_5d9daa3a6b964545be683e5110e0593b~mv2.webp/v1/fill/w_250,h_227,al_c,q_30,blur_30,enc_auto/c9b1c6_5d9daa3a6b964545be683e5110e0593b~mv2.webp)
![A framework for individualized splice-switching oligonucleotide therapy](https://static.wixstatic.com/media/c9b1c6_5d9daa3a6b964545be683e5110e0593b~mv2.webp/v1/fill/w_305,h_277,al_c,q_90,enc_auto/c9b1c6_5d9daa3a6b964545be683e5110e0593b~mv2.webp)
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...
212 views
![Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1](https://static.wixstatic.com/media/2be1ce_952266996fed46c58dddbc45f9b3e75c~mv2.png/v1/fill/w_305,h_172,fp_0.50_0.50,q_95,enc_auto/2be1ce_952266996fed46c58dddbc45f9b3e75c~mv2.webp)
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....
138 views
![Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic later](https://static.wixstatic.com/media/e164a5_990175c6c9454c8cbb4d86786b384aaf~mv2.png/v1/fill/w_306,h_278,fp_0.50_0.50,q_95,enc_auto/e164a5_990175c6c9454c8cbb4d86786b384aaf~mv2.webp)
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)...
39 views
![](https://static.wixstatic.com/media/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.jpg/v1/fill/w_250,h_141,al_c,q_30,blur_30,enc_auto/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.webp)
![New antisense oligonucleotide therapies reach first base in ALS](https://static.wixstatic.com/media/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.jpg/v1/fill/w_305,h_172,fp_0.50_0.50,q_90,enc_auto/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.webp)
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...
29 views
![](https://static.wixstatic.com/media/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.jpg/v1/fill/w_250,h_141,al_c,q_30,blur_30,enc_auto/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.webp)
![Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide](https://static.wixstatic.com/media/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.jpg/v1/fill/w_305,h_172,fp_0.50_0.50,q_90,enc_auto/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.webp)
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...
22 views
![OTS Rare Disease N-of-1+ Briefing Document, Jan 2020](https://static.wixstatic.com/media/e164a5_e9e7c9019f6b41898349d5e9931386fe~mv2.png/v1/fill/w_306,h_144,fp_0.50_0.50,q_95,enc_auto/e164a5_e9e7c9019f6b41898349d5e9931386fe~mv2.webp)
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...
53 views
![Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease](https://static.wixstatic.com/media/e164a5_af7c4a205b384635bb791f345867cbc7~mv2.png/v1/fill/w_305,h_243,fp_0.50_0.50,q_95,enc_auto/e164a5_af7c4a205b384635bb791f345867cbc7~mv2.webp)
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...
42 views
![](https://static.wixstatic.com/media/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.jpg/v1/fill/w_250,h_141,al_c,q_30,blur_30,enc_auto/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.webp)
![Drug Regulation in the Era of Individualized Therapies](https://static.wixstatic.com/media/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.jpg/v1/fill/w_305,h_172,fp_0.50_0.50,q_90,enc_auto/2be1ce_71e06761a2c842ffa87876a74df98b2f~mv2.webp)
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 ...
28 views
bottom of page