Kundaje: Even After Validation, Delivering AAV/CRISPR to Causal Cell Types Remains Unsolved

anshulkundaje · x · 2026-11-07

Continuing his thread on LLMs acting on noncoding variants, Stanford professor Anshul Kundaje notes that even if validation is solved, the therapeutic strategy remains non-trivial: delivery of AAV, CRISPR, small molecules or anything else to most causal cell types is unsolved. His earlier points covered how hard it is to run perturbation experiments in developmental cell types lacking accurate model systems, with organoids and iPSC-derived cells only partially viable.

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