How to create personalized gene editing platforms: Next steps toward interventional genetics

2025/10/31 by Rebecca C. Ahrens-Nicklas, Rebecca C. Ahrens‐Nicklas, Kiran Musunuru · 1 voice · 2 citations
Biochemistry, Genetics and Molecular Biology · #CRISPR and Genetic Engineering #Genomics and Rare Diseases #Virus-based gene therapy research

paper · pdf · doi:10.1016/j.ajhg.2025.10.006

openalex publication_date 2025/10/31 · openalex created_date 2025/10/31 · openalex updated_date 2026/07/27

Citations

• Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

Cited by

• Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform. [europepmc]
• Building CRISPR-Based Gene-Editing Platforms for Personalized Medicine: The Next Step in Interventional Genetics [europepmc]

Discussions

• Scaling personalized gene-editing therapy into a future of “interventional genetics” through “umbrella of umbrellas” master protocol clinical trial approaches doi.org/10.1016/j.aj... @ahrensnicklas.bs [bsky, 2 points, 0 comments]