University of California, San Francisco
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.
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…
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