NCT07396285|Recruiting
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Sponsor study identifier
observational
10,000
1 country
1
Study Start
May 1, 2026
Actual (reported)Primary Completion
December 2050
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
December 2050
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Gene editing approaches (including but not limited to: CRISPR, LNP, AAV, RNAi etc.) for the medical specialty of interventional genomics require (1) determination of the clinical relevance of genomic variants and (2) systematic evaluation of the 'editability' of those variants. Here, we seek to use in silico and in vitro analyses of genomic, cellular, and clinical data/specimens to (1) identify novel pathogenic variants underlying diseases and (2) examine the specificity and efficacy of various gene editing components across the entire human genomic landscape.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
December 2050
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
October 7, 2025
Actual (reported)February 9, 2026
Actual (reported)May 1, 2026
Actual (reported)December 2050
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
December 2050
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Improve our understanding of how specific mutations lead to dysfunction and cause disease
Up to 25 years
Empirically assess the edibility of individual participant genomic variants in silico and in patient-derived cells for the purpose of bespoke genomic therapies.
Up to 25 years
Support preclinical investigations of gene therapies specific to participants
Up to 25 years
Any individual diagnosed with a genetic disorder that may be amenable to gene therapy.
University of California, San Francisco
San Francisco, California, 94143, United States
Tsai SQ, Zheng Z, Nguyen NT, Liebers M, Topkar VV, Thapar V, Wyvekens N, Khayter C, Iafrate AJ, Le LP, Aryee MJ, Joung JK. GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat Biotechnol. 2015 Feb;33(2):187-197. doi: 10.1038/nbt.3117. Epub 2014 Dec 16.
Lazzarotto CR, Malinin NL, Li Y, Zhang R, Yang Y, Lee G, Cowley E, He Y, Lan X, Jividen K, Katta V, Kolmakova NG, Petersen CT, Qi Q, Strelcov E, Maragh S, Krenciute G, Ma J, Cheng Y, Tsai SQ. CHANGE-seq reveals genetic and epigenetic effects on CRISPR-Cas9 genome-wide activity. Nat Biotechnol. 2020 Nov;38(11):1317-1327. doi: 10.1038/s41587-020-0555-7. Epub 2020 Jun 15.
Tippi Mackenzie, MD
University of California, San Francisco
First Submitted
October 7, 2025
First Posted
February 9, 2026
Study Start
May 1, 2026
Actual (reported)Primary Completion
December 2050
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Contact the study team to confirm eligibility.
Ioannidis NM, Rothstein JH, Pejaver V, Middha S, McDonnell SK, Baheti S, Musolf A, Li Q, Holzinger E, Karyadi D, Cannon-Albright LA, Teerlink CC, Stanford JL, Isaacs WB, Xu J, Cooney KA, Lange EM, Schleutker J, Carpten JD, Powell IJ, Cussenot O, Cancel-Tassin G, Giles GG, MacInnis RJ, Maier C, Hsieh CL, Wiklund F, Catalona WJ, Foulkes WD, Mandal D, Eeles RA, Kote-Jarai Z, Bustamante CD, Schaid DJ, Hastie T, Ostrander EA, Bailey-Wilson JE, Radivojac P, Thibodeau SN, Whittemore AS, Sieh W. REVEL: An Ensemble Method for Predicting the Pathogenicity of Rare Missense Variants. Am J Hum Genet. 2016 Oct 6;99(4):877-885. doi: 10.1016/j.ajhg.2016.08.016. Epub 2016 Sep 22.
Study Completion
December 2050
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
September 1, 2026
Record last verified: 2026-08
De-identified specimens and data may be shared with other researchers
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Source: ClinicalTrials.gov.
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