Research for Individualized Therapeutics in Rare Genetic Disease

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 normal splicing, or return reading frame to an out-of-frame transcript to restore function, etc.) based on current acceptable understanding of ASO mechanisms of action and tissue/organ targeting efficiency. * Biological family member of an enrolled individual. * Would be able to travel to a Mayo Clinic site for ongoing treatment should a therapeutic be developed. * Treatment at the individual's current disease state would likely provide benefit based on current clinical data and understanding of the progression of the disease. -Or- * Biological family member of an enrolled individual * Able to provide informed consent or has a LAR available to provide informed consent Exclusion Criteria * Individuals who have situations that would limit compliance with the study requirements. * Institutionalized (i.e. Federal Medical Prison).