Genome Editing: Potential Treatment for Lysosomal Storage Diseases

• Published in: Current stem cell reports Vol. 1; no. 1; pp. 9 - 15
• Main Authors: de Carvalho, Talita Giacomet, da Silveira Matte, Ursula, Giugliani, Roberto, Baldo, Guilherme
• Format: Journal Article
• Language: English
• Published: Cham Springer International Publishing 01.03.2015; Springer Nature B.V
• Subjects: Animal models; Biomedical and Life Sciences; Biomedical Engineering/Biotechnology; Biomedicine; Cell Biology; CRISPR; Expression vectors; Fetuses; Gene Therapy; Genome Editing (SN Waddington; Genomes; Homology; Immunology; Life Sciences; Nuclease; Progenitor cells; Reviews; Section Editor; Stem Cells; Topical Collection on Genome Editing; Transcription activator-like effector nucleases; Zinc finger proteins; Zinc finger nucleases; TAL effector nucleases; Lysosomal storage disorders; CRISPR-Cas9
• ISSN: 2198-7866; 2198-7866
• DOI: 10.1007/s40778-014-0007-8

The recent progress in genome editing technology using the engineered zinc finger nucleases (ZFN), transcriptional activator-like effector nucleases (TALEN), and more recently, clustered regularly interspaced short palindromic repeat-CRISPR-associated protein 9 (CRISPR-Cas9) system have enabled the possibility of precisely modifying target sites in the genome. This technology brings hope of a cure for many genetic diseases. With this review, our goal is to discuss how targeted genome editing can be combined with hematopoietic stem cell transplantation and other approaches to be used for the treatment of a particular group of genetic diseases, the lysosomal storage disorders. We also highlight which diseases within this group would potentially benefit from this treatment and what are the main problems to be addressed to transform this promising technology into reality.