Recruiting

Interventional Genomics

Sponsor:

UCLA

Code:

NCT07396285

Conditions

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

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Accepted

Study Details

Brief 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

Study ID

NCT07396285

Start date

May 1, 2026

Status verified date

Aug, 2026

Completion date

Dec, 2050

Anticipated

Primary completion date

Dec, 2050

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Accepted

Inclusion Criteria:

For initial screening and discussion at huddle call:

  • Male and female individuals of any age, including pregnant women and their fetuses at any gestational age.
  • Self-referred or referred through their provider to the UCSF Center for Genome surgery/Interventional Genomics Board for a condition that is genetic or suspected to be genetic in origin.
  • UCSF patient OR consents to allow for their case to be presented at the IGB huddle.

For enrollment into registry or biobanking:

  • Any above participant that the IGB agrees would be appropriate for enrollment in the registry and biobanking portion of this protocol OR Any male or female family member of any age of someone enrolled in the registry and biobanking portion of this protocol.
  • Provides informed consent for participation in the registry and biobanking portion of the protocol.

Exclusion Criteria:

For initial screening and discussion at huddle call:

  • Individuals who have previously been discussed by the IGB and determined to not be appropriate to move forward into the registry and biobanking portion of the protocol, unless new information related to their case may change the initial assessment.
  • Individuals who are not impacted by a genetic or suspected genetic condition.

For enrollment into registry or biobanking:

-Any participant that the IGB agrees is inappropriate for enrollment in the registry and biobanking portion of this protocol.

Study Design

Enrollment

10000 participants

Anticipated

Interventions and Outcome Measures

Arms

Center for Genome Surgery Case Series Discussion

Enrollment in Registry and Biobank

Primary outcome measure

  • Improve our understanding of how specific mutations lead to dysfunction and cause disease [ Time Frame: 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. [ Time Frame: Up to 25 years ]

Central Contacts and Locations

Central contacts

Emma Canepa

(415) 476-7255Emma.Canepa@ucsf.edu

Locations

University of California, San Francisco

Recruiting

San Francisco, California, United States, 94143

Contacts

Emma Canepa

415-476-7255Emma.Canepa@ucsf.edu

More Information

Sponsor

University of California, San Francisco

Last update posted

Sep 1, 2026

Last verified

Aug, 2026

Keywords

  • Genetic
  • gene therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of California, San Francisco on 2026-09-01.