Mayo Clinic
The purpose of this research study is to identify individuals that have a rare genetic disease without an adequate therapeutic strategy that might be treatable with drug developed to target the disease-causing genetic alteration.
Inclusion Criteria: * Has Mayo Clinic or other medical health system ID, or another unique identifier. * Able to provide informed consent. * Individual must have evidence of a genetic disorder as determined by a provider or genetic counselor with causative or likely causative genetic variants identified by molecular testing. * Genetic variants must be hypothesized to be targetable using antisense oligonucleotide drugs (such as: knockdown gain of function alterations, increase protein production for reduced function alterations, or modulate mRNA splicing to correct abnormal splicing, promote n…
Patient phenotype and samples will be evaluated for individualized therapeutic drug development
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