Chromosome therapy: Potential strategies for the correction of severe chromosome aberrations

Large chromosomal aberrations occur commonly during development, resulting in complex and multisystem diseases. In spite of this high frequency, there are currently no means for correcting these disorders due to their complexity and involvement of multiple genes. Recently, several new approaches have been devised that target whole chromosomes in vitro, which are collectively referred to as “Chromosome Therapies.” These include silencing and selection for loss of the extra chromosome in trisomies, promotion of euploidy in an aneuploid culture, and forced loss and replacement of a chromosome. Here, we provide a review of Chromosome Therapy, and discuss potential directions for these methods clinically, as well as research applications and cellular models that can be made using these technologies. © 2016 Wiley Periodicals, Inc.

Source: American Journal of Medical Genetics Part C: Seminars in Medical Genetics - August 31, 2016 Category: Genetics & Stem Cells Authors: Kathleen Plona, Taehyun Kim, Katherine Halloran, Anthony Wynshaw ‐Boris Tags: Research Review Source Type: research