Precise Genome Modification Using Triplex Forming Oligonucleotides and Peptide Nucleic Acids

被引：3

作者：

Bahal, Raman ^{[1
]}

Gupta, Anisha ^{[1
]}

Glazer, Peter M. ^{[1
,2
]}

机构：

[1] Yale Univ, Sch Med, Dept Therapeut Radiol, New Haven, CT 06510 USA

[2] Yale Univ, Dept Genet, New Haven, CT 06510 USA

来源：

GENOME EDITING: THE NEXT STEP IN GENE THERAPY | 2016年 / 895卷

关键词：

TFOs; PNA; Recombination; Repair; Mutagenesis; Genome modification; NUCLEOTIDE EXCISION-REPAIR; INTRACHROMOSOMAL HOMOLOGOUS RECOMBINATION; WATSON-CRICK RECOGNITION; TARGETED GENE KNOCKOUT; DOUBLE-STRANDED DNA; DOUBLE-HELICAL DNA; MAMMALIAN-CELLS; MIXED-SEQUENCE; B-DNA; 2'-AMINOETHOXY-MODIFIED OLIGONUCLEOTIDES;

D O I：

10.1007/978-1-4939-3509-3_6

中图分类号：

Q [生物科学];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Many genetic disorders are caused by single base pair mutations which lead to defective protein synthesis. In addition to gene replacement therapy, modification of genomic DNA sequences at specific sites has been employed to manipulate the function and expression of various genes, which are implicated in various genetic disorders. On this front, triplex technology has been used to alter the expression of different genes by correcting mutations site specifically via homologous recombination (HR) or targeted mutagenesis based mechanisms. In this chapter we will discuss the advances made in triplex technology involving triplex forming oligonucleotides (TFOs) and peptide nucleic acids (PNAs) for site specific genome editing.

引用

页码：93 / 110

页数：18

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