Main
This registry is divided into three parts. That can be accessed via the buttons on the main page. The search function on the main page allows searching in all registries.
1. N-of-1 Projects
This part of the registry lists ongoing n-of-1 and n-of-few projects by N1C members as well as collaborators and further organizations. The projects are listed alphabetically by gene name.
Each project can be opened as its own entry, containing additional information on the variant, status of the project, availability of Investigational New Drug Application (IND),
and cell lines/animal models. Additionally, the entry pages also contain contact information for the lab and PI to reach out to. The search function
will search the full registry, including parts of the entries that are not visible in the main table.
2. Marketed Genetic Therapies
This registry contains the Marketed Genetic Therapies available for larger groups of patients. The overview table is sorted alphabetically by marketed drug name.
No single entries are available here. The search function will search through all marketed therapies.
3. Assessed Variants
This registry contains variants assessed for their eligibility for a therapeutic intervention. The variants are listed alphabetically. Each variant entry can be opened to receive more information on the assessment. Assessments are performed by trained assessors under lead of Dr. Charu Kaiwar.
For questions, suggestions, or corrections, please contact us at:
generegistry@n1collaborative.org
n-Lorem Portfolio: n-Lorem is an independent non-profit organisation developing antisense oligonucleotides. For more information on their ASOs, please reach out to n-Lorem directly: www.nlorem.org
For other therapies targeting genetic diseases, please also visit: RX Genes or Treatabolome DB
For an overview of ongoing clinical trials for genetic therapies, please visit: Clinical Trials - Gene Therapy Trial Browser
This registry is provided by the N=1 Collaborative and run by the Patient Identification Working Group. We want to thank all contributors to this registry, especially Kailee Yap and Andy Drackley (Center for Genomics, Ann & Robert H. Lurie Children’s Hospital of Chicago) who maintain the registry, as well as David Cheerie (SickKids, Toronto), Nicole Nolen (N=1 Collaborative), Marlen Lauffer (Dutch Center for RNA Therapeutics, Leiden University Medical Center), and the members of the variant assessment task force.