Genome Editing for Mucopolysaccharidoses

被引:29
作者
Poletto, Edina [1 ,2 ]
Baldo, Guilherme [1 ,2 ]
Gomez-Ospina, Natalia [3 ]
机构
[1] Hosp Clin Porto Alegre, Gene Therapy Ctr, BR-90035007 Porto Alegre, RS, Brazil
[2] Univ Fed Rio Grande do Sul, Postgrad Program Genet & Mol Biol, BR-91501970 Porto Alegre, RS, Brazil
[3] Stanford Univ, Dept Pediat, Stanford, CA 94305 USA
关键词
genome editing; gene therapy; mucopolysaccharidoses; lysosomal storage disease; CRISPR; Cas9; Hurler; Hunter; Zinc Finger Nucleases; viral vectors; non-viral vectors; hematopoietic stem cell transplantation; ENZYME-REPLACEMENT THERAPY; HEMATOPOIETIC-CELL TRANSPLANTATION; TERM-FOLLOW-UP; GENE-THERAPY; IN-VIVO; STORAGE DISEASES; OPEN-LABEL; CAS9; EFFICACY; OUTCOMES;
D O I
10.3390/ijms21020500
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Genome editing holds the promise of one-off and potentially curative therapies for many patients with genetic diseases. This is especially true for patients affected by mucopolysaccharidoses as the disease pathophysiology is amenable to correction using multiple approaches. Ex vivo and in vivo genome editing platforms have been tested primarily on MSPI and MPSII, with in vivo approaches having reached clinical testing in both diseases. Though we still await proof of efficacy in humans, the therapeutic tools established for these two diseases should pave the way for other mucopolysaccharidoses. Herein, we review the current preclinical and clinical development studies, using genome editing as a therapeutic approach for these diseases. The development of new genome editing platforms and the variety of genetic modifications possible with each tool provide potential applications of genome editing for mucopolysaccharidoses, which vastly exceed the potential of current approaches. We expect that in a not-so-distant future, more genome editing-based strategies will be established, and individual diseases will be treated through multiple approaches.
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页数:20
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