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https://www.academia.edu/8955106/Trans-splicing_ribozymes_for_targeted_gene_delivery1
Trans-splicing ribozymes for targeted gene delivery1
https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(04)00172-8
A variety of mutations in the p53 tumor suppressor gene have been found in over half of human tumors. Thus, restoration of wild-type p53 activity by repair of mutant RNA has been previously suggested as an approach to cancer treatment. To explore the potential utility of RNA repair for cancer therapy, we developed a group I intron-based ribozyme that can replace mutant p53 RNA with a wild-type ...
https://link.springer.com/article/10.1007/s10529-005-2883-6
Current gene therapy protocols against cancer often have limited target specificity. Here, a novel tumor-specific targeted gene delivery procedure, which is based on Tetrahymena group I intron ribozyme, is presented. This ribozyme can target a cancer-specific transcript and then replace the RNA with new transcripts, resulting in induction of the transgene activity selectively in cancer cells ...
https://www.researchgate.net/publication/32010852_313_Efficacy_of_Group_I_Intron-Based_Trans-Splicing_Ribozyme_in_Tumor-Targeted_Gene_Therapy
Group I intron-based trans-splicing ribozyme, which can specifically reprogram human telomerase reverse transcriptase (hTERT) RNA, could be a useful tool for tumor-targeted gene therapy.
https://www.researchgate.net/publication/241604616_615_Feasibility_of_hTERT-targeting_trans-splicing_ribozyme_in_tumor-targeted_gene_therapy
In the present study, we describe a new tumor-targeted gene therapy tool that is based upon trans-splicing ribozymes. Trans-splicing ribozymes are RNA-based catalysts capable of performing both ...
https://febs.onlinelibrary.wiley.com/doi/full/10.1016/j.febslet.2006.08.021
Aug 22, 2006 · Cancer‐selective induction of cytotoxicity by tissue‐specific expression of targeted trans‐splicing ribozyme ... development of control system of transgene expression combined with tissue‐specific gene delivery systems should be considered. ... Higher expression of trans‐splicing ribozymes was observed to enhance level of RNA repair ...
https://clincancerres.aacrjournals.org/content/14/1/281
Conclusions: This study shows that systemically delivered adenovirus harboring trans -splicing ribozyme can recognize cancer-specific transcripts and reprogram them to combat the cancer cells. Use of trans -splicing ribozymes seems to be a potentially useful gene therapy for cancer.
https://www.jci.org/articles/view/16481
RNA repair has been proposed as a novel gene-based therapeutic strategy. Modified Tetrahymena group I intron ribozymes have been used to mediate trans-splicing of therapeutically relevant RNA transcripts, but the efficiency of the ribozyme-mediated RNA repair process has not been determined precisely and subsequent restoration of protein function has been demonstrated only by indirect means.
https://academic.oup.com/nar/article/30/24/e141/1077921
Dec 15, 2002 · Abstract. Trans‐splicing ribozymes are RNA‐based catalysts capable of splicing RNA sequences from one transcript specifically into a separate target transcript.In doing so, a chimeric mRNA can be produced, and new gene activities triggered in living cells dependent on …
https://patents.justia.com/patent/10557140
The present invention relates to a recombinant vector, characterized by including a cytotoxic T-lymphocyte-associated protein-4 (CTLA-4)-targeting trans-splicing ribozyme expression cassette for delivery of chimeric antigen receptor, wherein the expression cassette includes: (i) a CTLA-4-targeting trans-splicing ribozyme; and (ii) a polynucleotide encoding a chimeric antigen receptor ligated ...
https://patents.google.com/patent/KR101153844B1/en
PURPOSE: A trans-splicing group I ribozyme is provided to specifically recognize a base mutated gene transcript and to treat and diagnose cancer cells. CONSTITUTION: A trans-splicing group I ribozyme which specifically recognizes Kras G12V RNA is AS100KRib2 of sequence number 27. The ribozyme treats or diagnoses cancer cells only by selectively inducing apoptosis of cancer cells.
https://www.pnas.org/content/pnas/96/7/3507.full.pdf
These ribozymes might be useful for the selective delivery of new gene activities in virus-infected or malignant cells. For example, trans-splicing ribozymes could provide the basis for a virus resistance strategy in plants. Trans-splicing ribozymes could be designed to …
https://patents.google.com/patent/CA2100068A1/en
The design of new ribozymes capable of self-catalyzed trans-splicing which are based upon the catalytic core of a Group I intron are described. Using this design, it is possible to construct ribozymes capable of efficiently splicing a new 3' exon sequence into any chosen target RNA sequence in a highly precise manner. Inactive pro-ribozyme forms are also described.
https://www.jci.org/articles/view/19386
Group I introns are ribozymes that carry out two transesterification reactions in order to excise themselves from a precursor transcript ().The self-splicing reaction pathway of the Tetrahymena intron is illustrated in Figure 1.The 5′ splice site is located in helix P1, which is formed by base-pairing between the exon sequence and the internal guide sequence (IGS) of the intron.
https://www.advancedsciencenews.com/therapeutic-applications-group-intron-based-trans-splicing-ribozymes/
In a recently published review article in WIREs RNA, “Therapeutic applications of group I intron-based trans-splicing ribozymes,” Lee et al. reviews and discusses the current status of the trans-splicing ribozyme as an emerging gene therapeutic approach with high efficacy and specificity.
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