University of California, San Francisco
San Francisco, California, 94143, United States
Not Yet Recruiting
NCT Number: NCT07396285
UCSF Center for Genome Surgery Biobank and Registry
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.
Not Yet Recruiting
Trial opening soon.
Get NotifiedKey information
Sex eligibility
All sexes
Study type
Observational
Primary location
Who can participate
Healthy volunteers accepted: Yes
Only the study team can determine whether someone qualifies for participation.
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.
Treatment and study plan
Primary outcomes
-
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
Other outcomes
-
Support preclinical investigations of gene therapies specific to participants
Time frame: Up to 25 years
Study contacts
Contact information is provided by the study sponsor or research team.
Sponsors and collaborators
Lead sponsor
University of California, San Francisco
Other
Registry information
Important dates
- Study start
- 2026
- Primary completion
- 2050
- Study completion
- 2050
- First posted
- Feb 9, 2026
- Registry last updated
- Feb 9, 2026
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.