Publications

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...

Publications

The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap

Publications

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...

Publications

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 )...

Publications

A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides

Publications

Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing

Publications

How to pay for individualized genetic medicines

Publications

Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments

Publications

Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

Publications

A Multistakeholder Perspective on Advancing Individualized Therapeutics

Publications

A framework for individualized splice-switching oligonucleotide therapy

Publications

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1

Publications

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic later

Publications

New antisense oligonucleotide therapies reach first base in ALS

Publications

Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

Publications

OTS Rare Disease N-of-1+ Briefing Document, Jan 2020

Publications

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

Publications

A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases

The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap

N-of-1 Studies in an Era of Precision Medicine

Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics

A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides

Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing

How to pay for individualized genetic medicines

Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments

Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

A Multistakeholder Perspective on Advancing Individualized Therapeutics

A framework for individualized splice-switching oligonucleotide therapy

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic later

New antisense oligonucleotide therapies reach first base in ALS

Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

OTS Rare Disease N-of-1+ Briefing Document, Jan 2020

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

Drug Regulation in the Era of Individualized Therapies

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