Authors: Bruce A Bunnell; Reza Izadpanah; Harry C Ledebur Jr; Carl F Perez

Source: Expert Opinion on Biological Therapy, Volume 5, Number 2, 1 February 2005 , pp. 195-206(12)

Publisher: Informa Healthcare

Abstract:

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 denovo 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 exvivo 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 exvivo gene therapy treatment of lysosomal storage diseases, specifically Sandhoff (MIM #268800) and Krabbe (MIM #245200) diseases.

Keywords: artificial chromosomes; gene therapy; Krabbe disease; lysosomal storage disease; Sandhoff disease; stem cells

Document Type: Review article

DOI: http://dx.doi.org/10.1517/14712598.5.2.195

Publication date: 2005-02-01

More about this publication?

• Editorial Board
• Information for Authors
• Subscribe to this Title
• Terms & Conditions
• ingentaconnect is not responsible for the content or availability of external websites

Related content

• In this: publication
• By this: publisher
• In this Subject: Organic Chemistry ,  Oncology
• By this author: Bruce A Bunnell ;  Reza Izadpanah ;  Harry C Ledebur Jr ;  Carl F Perez

Website © Publishing Technology. Article copyright remains with the publisher, society or author(s) as specified within the article.

• Terms and conditions
• Privacy policy
• Information for advertisers