Rare Genetic Disorders: Novel Treatment Strategies and Insights Into Human Biology.

ABSTRACT: The last decade has seen a dramatic increase in innovative ideas for the treatment of genetic disorders for which no curative therapies exist. Gene and protein replacement therapies stand out as novel approaches to treat a select group of these diseases, such as certain tissue fragility disorders. Further, the advent of stem cell approaches, such as induced pluripotent stem cells (iPSC) technology, has led to the development of new methods of creating replacement tissues for regenerative medicine. This coincided with the discovery of genome editing techniques, which allow for the correction of disease-causing mutations. The culmination of these discoveries suggests that new and innovative therapies for monogenetic disorders affecting single organs or tissues are on the horizon. Challenges

SUBMITTER: Koch PJ 

PROVIDER: S-EPMC8378213 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature