UCSF Center for Genome Surgery Biobank and Registry

NCT07396285 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 10000

Last updated 2026-02-09

No results posted yet for this study

Summary

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.

Conditions

  • Any Conditions That Are Genetic or Suspected to be Genetic in Origin

Sponsors & Collaborators

Principal Investigators

  • Tippi Mackenzie, MD · University of California, San Francisco

Eligibility

Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2026-05-01
Primary Completion
2050-12-31
Completion
2050-12-31

Countries

  • United States

Study Locations

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Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07396285 on ClinicalTrials.gov