Current dual AAV vector approaches have important limitations, e.g., low reconstitution efficiency, production of alien proteins, or low flexibility in split site selection. Here, we present a dual AAV vector technology based on reconstitution via mRNA trans-splicing (REVeRT). REVeRT is flexible ...
近日,来自瑞士苏黎世大学的Elvir Becirovic实验室在《Nature Communications》上发表文章‘mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy’,作者提出了一种基于mRNA反式剪接重组的双AAV载体技术(reconstitution via mRNA trans-splicing ,REVeRT)。为了测试和优化REVeRT,作者建立了荧光...
trans-splicing occurs between one of two downstream splice donors in the sequence encoding a C-terminal V5 epitope tag and an upstream splice acceptor in the 5′ region of the pCS2(+) host plasmid. Using deletion and fusion
Overall, these results confirm that CRAFT can edit endogenous mRNA tran- scripts and is amenable to delivery by AAV vectors. To determine the absolute trans-splicing efficiency of CRAFT, we replaced the fragments of EGFP in either rcRNA with either exons 1–10 or exons 11–12 of LMNA in ...
Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise, however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal muscle tissues. In general, AAV vectors are used for in vivo delivery, but AAV injections cannot be repeated because of neutralizati...
Molec- ular analysis of mutations affecting hprt mRNA splicing in human T-lym- phocytes in vivo. Environ. Mol. Mutagen. 19:7-13.Rossi AM , Tates AD , van Zeeland AA , Vrieling H ( 1992 ) Molecular analysis of mutations affecting hprt mRNA splicing in human T-lymphocytes in vivo . ...
LAI Y,DUAN D.Optimizing intron splicing signal overcomes the mRNA accumulation barrier in trans-splicing AAV vectors. Molecular Therapy . 2006LAI Y, DUAN D. Optimizing intron splicing signal overcomes the mRNA accumulation barrier in trans-splicing AAV vectors [ J ]. Mol Ther, 2006, 13: 407-...
mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy. Nat. Commun. 2023, 14, 6578. [Google Scholar] [CrossRef] [PubMed] Gai, C.; Pomatto, M.A.C.; Deregibus, M.C.; Dieci, M.; Piga, A.; Camussi, G. Edible Plant-Derived Extracellular Vesicles for ...
The latter, has demonstrated the potential to deliver translational therapeutic concentration levels of proteinic constructs and superiority of safety when compared to viral vectors and/or DNA plasmid transfection, due its transient nature [19]. Elaborating on this, the risk of insertional mutagenesis ...