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- W3124160933 endingPage "166" @default.
- W3124160933 startingPage "145" @default.
- W3124160933 abstract "Genetic diseases cause numerous complex and intractable pathologies. DNA sequences encoding each human's complexity and many disease risks are contained in the mitochondrial genome, nuclear genome, and microbial metagenome. Diagnosis of these diseases has unified around applications of next-generation DNA sequencing. However, translating specific genetic diagnoses into targeted genetic therapies remains a central goal. To date, genetic therapies have fallen into three broad categories: bulk replacement of affected genetic compartments with a new exogenous genome, nontargeted addition of exogenous genetic material to compensate for genetic errors, and most recently, direct correction of causative genetic alterations using gene editing. Generalized methods of diagnosis, therapy, and reagent delivery into each genetic compartment will accelerate the next generations of curative genetic therapies. We discuss the structure and variability of the mitochondrial, nuclear, and microbial metagenomic compartments, as well as the historical development and current practice of genetic diagnostics and gene therapies targeting each compartment." @default.
- W3124160933 created "2021-02-01" @default.
- W3124160933 creator A5067971060 @default.
- W3124160933 creator A5070915778 @default.
- W3124160933 date "2021-01-24" @default.
- W3124160933 modified "2023-10-17" @default.
- W3124160933 title "Genetic Disease and Therapy" @default.
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