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.jpg): Failed to open stream: No such file or directory in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 117 Hum+Genet
2016 ; 135
(9
): 1059-70
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TALEN gene editing takes aim on HIV
#MMPMID27170155
Benjamin R
; Berges BK
; Solis-Leal A
; Igbinedion O
; Strong CL
; Schiller MR
Hum Genet
2016[Sep]; 135
(9
): 1059-70
PMID27170155
show ga
Transcription activator-like effector nucleases (TALENs) are one of several types
of programmable, engineered nucleases that bind and cleave specific DNA
sequences. Cellular machinery repairs the cleaved DNA by introducing indels. In
this review, we emphasize the potential, explore progress, and identify
challenges in using TALENs as a therapeutic tool to treat HIV infection. TALENs
have less off-target editing and can be more effective at tolerating HIV escape
mutations than CRISPR/Cas-9. Scientists have explored TALEN-mediated editing of
host genes such as viral entry receptors (CCR5 and CXCR4) and a protein involved
in proviral integration (LEDGF/p75). Viral targets include the proviral DNA,
particularly focused on the long terminal repeats. Major challenges with
translating gene therapy from bench to bedside are improving cleavage efficiency
and delivery, while minimizing off-target editing, cytotoxicity, and
immunogenicity. However, rapid improvements in TALEN technology are enhancing
cleavage efficiency and specificity. Therapeutic testing in animal models of HIV
infection will help determine whether TALENs are a viable HIV treatment therapy.
TALENs or other engineered nucleases could shift the therapeutic paradigm from
life-long antiretroviral therapy toward eradication of HIV infection.
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