Development of mammalian artificial chromosomes for the treatment of genetic diseases: Sandhoff and Krabbe diseases

Bruce A. Bunnell, Reza Izadpanah, Harry C. Ledebur, Carl F. Perez

Research output: Contribution to journalReview articlepeer-review

4 Scopus citations


Mammalian artificial chromosomes (MACs) are being developed as alternatives to viral vectors for gene therapy applications, as they allow for the introduction of large payloads of genetic information in a non-integrating, autonomously replicating format. One class of MACs, the satellite DNA-based artificial chromosome expression vehicle (ACE), is uniquely suited for gene therapy applications, in that it can be generated de novo in cells, along with being easily purified and readily transferred into a variety of recipient cell lines and primary cells. To facilitate the rapid engineering of ACEs, the ACE System was developed, permitting the efficient and reproducible loading of pre-existing ACEs with DNA sequences and/or target gene(s). As a result, the ACE System and ACEs are unique and versatile platforms for ex vivo gene therapy strategies that circumvent and alleviate existing safety and delivery limitations surrounding conventional gene therapy vectors. This review will focus on the status of MAC technologies and, in particular, the application of the ACE System towards an ex vivo gene therapy treatment of lysosomal storage diseases, specifically Sandhoff (MIM #268800) and Krabbe (MIM #245200) diseases.

Original languageEnglish
Pages (from-to)195-206
Number of pages12
JournalExpert Opinion on Biological Therapy
Issue number2
StatePublished - Feb 2005


  • Artificial chromosomes
  • Gene therapy
  • Krabbe disease
  • Lysosomal storage disease
  • Sandhoff disease
  • Stem cells


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