scholarly journals Modifying inter-cistronic sequence significantly enhances IRES dependent second gene expression in bicistronic vector: Construction of optimised cassette for gene therapy of familial hypercholesterolemia

2019 ◽  
Vol 4 (1) ◽  
pp. 1-14 ◽  
Author(s):  
Faisal A. Al-Allaf ◽  
Zainularifeen Abduljaleel ◽  
Mohammad Athar ◽  
Mohiuddin M. Taher ◽  
Wajahatullah Khan ◽  
...  
2019 ◽  
Vol 19 (4) ◽  
pp. 255-263 ◽  
Author(s):  
Yuangang Wu ◽  
Xiaoxi Lu ◽  
Bin Shen ◽  
Yi Zeng

Background: Osteoarthritis (OA) is a disease characterized by progressive degeneration, joint hyperplasia, narrowing of joint spaces, and extracellular matrix metabolism. Recent studies have shown that the pathogenesis of OA may be related to non-coding RNA, and its pathological mechanism may be an effective way to reduce OA. Objective: The purpose of this review was to investigate the recent progress of miRNA, long noncoding RNA (lncRNA) and circular RNA (circRNA) in gene therapy of OA, discussing the effects of this RNA on gene expression, inflammatory reaction, apoptosis and extracellular matrix in OA. Methods: The following electronic databases were searched, including PubMed, EMBASE, Web of Science, and the Cochrane Library, for published studies involving the miRNA, lncRNA, and circRNA in OA. The outcomes included the gene expression, inflammatory reaction, apoptosis, and extracellular matrix. Results and Discussion: With the development of technology, miRNA, lncRNA, and circRNA have been found in many diseases. More importantly, recent studies have found that RNA interacts with RNA-binding proteins to regulate gene transcription and protein translation, and is involved in various pathological processes of OA, thus becoming a potential therapy for OA. Conclusion: In this paper, we briefly introduced the role of miRNA, lncRNA, and circRNA in the occurrence and development of OA and as a new target for gene therapy.


2001 ◽  
Vol 287 (4) ◽  
pp. 1034-1040 ◽  
Author(s):  
Hiroki Ishikawa ◽  
Keisuke Nakata ◽  
Fumihiro Mawatari ◽  
Toshihito Ueki ◽  
Shotaro Tsuruta ◽  
...  

2013 ◽  
Vol 12 (1) ◽  
pp. 373 ◽  
Author(s):  
Jeffrey C Wagner ◽  
Stephen J Goldfless ◽  
Suresh M Ganesan ◽  
Marcus CS Lee ◽  
David A Fidock ◽  
...  

1999 ◽  
Vol 380 (6) ◽  
Author(s):  
H. Büeler

AbstractAdeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression


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