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Articles & Podcasts

A Baby Receives the First Customized CRISPR Treatment

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...

Articles & Podcasts

'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis

Splice-Altering Transposon Insertions in Neurodegenerative Diseases and Its Application in an Individualized ASO Therapy for Retinitis Pigmentosa

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

Preclinical development of personalized ASO therapeutics using patient-derived organoid systems

Towards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders

Workshops & Presentations

Navigating Institutional Implementation: A Quarterly Open Forum for N-of-1 Clinical Trials

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