UCSF Center for Genome Surgery Biobank and Registry for Genetic Conditions

This study is creating a collection of information and samples (a biobank and registry) to better understand genetic conditions. It's an observational study, meaning researchers will collect data without giving you a specific treatment. The goal is to learn how changes in your genes (mutations) cause illness and how effective different gene editing tools like CRISPR, LNP, AAV, and RNAi might be in treating these conditions. You can join if you have a condition that is genetic or thought to be genetic. This study aims to improve our understanding of genetic diseases and how to develop future gene therapies over a long period, up to 25 years.

Study design: This is an observational study planning to enroll up to 10,000 participants. It is not a treatment study, but rather a collection of information and samples.
What's involved: Not specified in the trial record.
Compensation: Not stated in the trial record.
Follow-up: The study aims to improve understanding and assess gene editing over a long period, up to 25 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

UCSF Center for Genome Surgery Biobank and Registry

Not Yet Recruiting

Not specifiedAll AgesObservational

University of California, San Francisco

~10,000 participants

Updated 2026-02-09 on ClinicalTrials.gov

At a glance

Recruiting sites

0 of 1 listed site is recruiting right now

RecruitingSuspended, closed, or not yet open

What they're measuring

Improve our understanding of how specific mutations lead to dysfunction and cause disease

Measured over Up to 25 years

+1 more outcome measured

Conditions

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

Where it's being run

1 sites across 1 states

California1

Study leadership

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

Who to contact

Emma Canepa

[email protected]

(415) 476-7255

Email the study team

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Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

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Eligibility criteria

Inclusion

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.

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

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.

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

What this trial measures

Improve our understanding of how specific mutations lead to dysfunction and cause diseaseUp 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

Trialytics