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2016 ; 17
(5
): ä Nephropedia Template TP
gab.com Text
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English Wikipedia
In Vivo Delivery Systems for Therapeutic Genome Editing
#MMPMID27128905
Wang L
; Li F
; Dang L
; Liang C
; Wang C
; He B
; Liu J
; Li D
; Wu X
; Xu X
; Lu A
; Zhang G
Int J Mol Sci
2016[Apr]; 17
(5
): ä PMID27128905
show ga
Therapeutic genome editing technology has been widely used as a powerful tool for
directly correcting genetic mutations in target pathological tissues and cells to
cure of diseases. The modification of specific genomic sequences can be achieved
by utilizing programmable nucleases, such as Meganucleases, zinc finger nucleases
(ZFNs), transcription activator-like effector nucleases (TALENs), and the
clustered regularly-interspaced short palindromic repeat-associated nuclease Cas9
(CRISPR/Cas9). However, given the properties, such as large size, negative
charge, low membrane penetrating ability, as well as weak tolerance for serum,
and low endosomal escape, of these nucleases genome editing cannot be
successfully applied unless in vivo delivery of related programmable nucleases
into target organisms or cells is achieved. Here, we look back at delivery
strategies having been used in the in vivo delivery of three main genome editing
nucleases, followed by methodologies currently undergoing testing in clinical
trials, and potential delivery strategies provided by analyzing characteristics
of nucleases and commonly used vectors.