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Investigating REPAIRv2 as a Tool to Edit CFTR mRNA with Premature Stop Codons.

Abstract：

:Cystic fibrosis (CF) is caused by mutations in the gene encoding the transmembrane conductance regulator (CFTR) protein. Some CF patients are compound heterozygous or homozygous for nonsense mutations in the CFTR gene. This implies the presence in the transcript of premature termination codons (PTCs) responsible for a truncated CFTR protein and a more severe form of the disease. Aminoglycoside and PTC124 derivatives have been used for the read-through of PTCs to restore the full-length CFTR protein. However, in a precision medicine framework, the CRISPR/dCas13b-based molecular tool "REPAIRv2" (RNA Editing for Programmable A to I Replacement, version 2) could be a good alternative to restore the full-length CFTR protein. This RNA editing approach is based on the targeting of the deaminase domain of the hADAR2 enzyme fused to the dCas13b protein to a specific adenosine to be edited to inosine in the mutant mRNA. Targeting specificity is allowed by a guide RNA (gRNA) complementarily to the target region and recognized by the dCas13b protein. Here, we used the REPAIRv2 platform to edit the UGA PTC to UGG in different cell types, namely IB3-1 cells, HeLa, and FRT cells engineered to express H2BGFP opal and CFTR W1282X , respectively.

journal_name

Int J Mol Sci

journal_title

International journal of molecular sciences

authors

Melfi R,Cancemi P,Chiavetta R,Barra V,Lentini L,Di Leonardo A

doi

10.3390/ijms21134781

subject

Has Abstract

pub_date

2020-07-06 00:00:00

issue

13

issn

1422-0067

pii

ijms21134781

journal_volume

21

pub_type

杂志文章

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