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
“Using Lipid Nanoparticles to Efficiently Deliver
CRISPR/Cas9 for Genome Editing”
The 15th Annual Meeting of the Oligonucleotide Therapeutics Society
Oct. 14, 2019
“In Silico, Biochemical and Cell-based Integrative Genomics Identifies Precise CRISPR / Cas9 Targets for Human Therapeutics”
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct. 23, 2019
“CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria”
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct. 24, 2019
“In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression”
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct. 25, 2019
Engineered Cell Therapy
“Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout”
European Society of Gene and Cell Therapy 27th Annual Meeting
Oct. 24, 2019
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