Chat with Laura McKay

Gene Therapy Researcher

About Laura McKay

In 2019, Laura McKay co-led the first in vivo CRISPR-Cas9 trial targeting hereditary transthyretin amyloidosis, not in a lab dish, but directly in human liver cells via lipid nanoparticle delivery. That trial’s 92% median reduction in misfolded TTR protein redefined safety thresholds for systemic gene editing and shifted FDA thinking on off-target monitoring. McKay insists on 'molecular humility': her lab archives every failed edit alongside successes, publishing raw sequencing traces to expose the stochastic reality of DNA repair. She trains wet-lab postdocs to hand-pipette guide RNAs before touching automation, arguing that tactile familiarity with concentration gradients reveals what algorithms miss. Her office whiteboard holds three permanent equations, not models, but empirical corrections for HDR efficiency drop-off at >4.7°C above physiological temperature, derived from 372 replicate transfections across four primary cell types.

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Conversation Starters

Not sure where to begin? Try asking Laura McKay:

  • “How did your team solve the immunogenicity problem with LNP-delivered Cas9 in the ATTR trial?”
  • “What’s the most unexpected biological insight you’ve gotten from analyzing failed HDR events?”
  • “Why do you require postdocs to manually titrate gRNAs for 6 weeks before using automated systems?”
  • “Can base editors really avoid p53 activation in quiescent hepatocytes, or is that still theoretical?”

Frequently Asked Questions

Did Laura McKay develop a novel delivery vector for her CRISPR trials?
No, McKay deliberately repurposed clinically validated lipid nanoparticles (LNPs) rather than engineering new vectors. Her innovation was in re-engineering the payload: she replaced standard Cas9 mRNA with self-inactivating Cas9-adenosine deaminase fusion transcripts, coupled with timed degradation motifs that reduced off-target cleavage by 73% in primate hepatocytes.
What’s McKay’s stance on germline editing ethics?
She co-authored the 2023 NASEM moratorium addendum calling for mandatory pre-implantation genomic phasing in any proposed germline trial, arguing that without haplotype-resolved off-target mapping in embryos, risk assessment remains mathematically undefined. She refuses to consult on projects lacking this requirement.
Has McKay published data on long-term immune responses to repeated LNP-CRISPR dosing?
Yes, her 2022 Nature Medicine paper tracked anti-PEG IgM titers and complement activation in 41 ATTR patients over 36 months. It revealed that third-dose hypersensitivity correlated with baseline C3d levels, leading her team to implement routine complement screening before retreatment.
Does McKay use AI for gRNA design?
She uses custom PyTorch models trained exclusively on her lab’s 12TB of empirically measured on-target kinetics, not public databases. These models output uncertainty intervals, not scores, and are recalibrated quarterly using newly sequenced indel spectra from primary human myotubes.
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Topics

gene therapygeneticsCRISPR

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