What this study is
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.
Study details
- Sponsor
- University of California, San Francisco
- Conditions
- Any Conditions That Are Genetic or Suspected to be Genetic in Origin
- Enrolling
- 10,000 people
- Starts
- 2026-05-01
- Est. completion
- 2050-12
- Record
- View on ClinicalTrials.gov ↗
Who can join
Sex ALLHealthy volunteers AcceptedYou may qualify if
- 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.
You can't join if
- 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.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (1)
1 openUniversity of California, San Francisco
San Francisco, California, United States
- Emma Canepa · Contact415-476-7255Emma.Canepa@ucsf.edu
Recruiting
Study team
Sponsor
University of California, San Francisco
Study ID UCSF_CGS_Biobank_Registry
Principal investigator
Tippi Mackenzie, MD
University of California, San Francisco
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.