In vivo
“A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing”
Cell Reports
Feb. 27, 2018
“Rescue of Amyloid Deposition Phenotype After Single-Treatment CRISPR/Cas9 Gene Editing in a Humanized Mouse Model of ATTR”
American Society of Gene and Cell Therapy 21st Annual Meeting
May 16, 2018
“Development of NTLA-1001: First-in-Class, LNP-CRISPR/Cas9 Mediated Genome Editing Therapeutic for the Treatment of ATTR”
Genome Engineering: The CRISPR-Cas Revolution at Cold Spring Harbor Laboratory Presentation
Aug. 23, 2018
“Delivering on the therapeutic potential of CRISPR/Cas9: Development of an LNP-mediated genome editing therapeutic for the treatment of ATTR”
26th Annual Congress of the European Society of Gene and Cell Therapy
Oct. 18, 2018
“Supra-therapeutic levels of transgene expression achieved in vivo by CRISPR/Cas9 mediated targeted gene insertion”
26th Annual Congress of the European Society of Gene and Cell Therapy
Oct. 18, 2018
“CRISPR/Cas9-Mediated Targeted Insertion of Human F9 Achieves Therapeutic Circulating Protein Levels in Mice and Non-Human Primates”
American Society of Gene and Cell Therapy 22nd Annual Meeting
Apr. 29, 2019
“CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria”
American Society of Gene and Cell Therapy 22nd Annual Meeting
May 2, 2019
“Development of NTLA-2001, a CRISPR/Cas9 Genome Editing Therapeutic for the Treatment of ATTR”
2nd European Congress for ATTR Amyloidosis
Sept. 1, 2019
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