scholarly journals Synchronous conjugation of i-motif DNA and therapeutic siRNA on the vertexes of tetrahedral DNA nanocages for efficient gene silence

Author(s):  
Xiu Han ◽  
Xiang Xu ◽  
Ziheng Wu ◽  
Zhenghong Wu ◽  
Xiaole Qi
Keyword(s):  
Diabetes ◽  
1996 ◽  
Vol 45 (9) ◽  
pp. 1197-1203 ◽  
Author(s):  
J. Saldeen ◽  
D. T. Curiel ◽  
D. L. Eizirik ◽  
A. Andersson ◽  
E. Strandell ◽  
...  

Author(s):  
Xin Gou ◽  
Hua-An Yang ◽  
Wei-Yang He ◽  
Ming-Chao Xioa ◽  
Ming Wang

2020 ◽  
Vol 20 (5) ◽  
pp. 321-332
Author(s):  
Yunbo Liu ◽  
Xu Zhang ◽  
Lin Yang

Adeno-associated virus (AAV) is a promising vector for in vivo gene therapy because of its excellent safety profile and ability to mediate stable gene expression in human subjects. However, there are still numerous challenges that need to be resolved before this gene delivery vehicle is used in clinical applications, such as the inability of AAV to effectively target specific tissues, preexisting neutralizing antibodies in human populations, and a limited AAV packaging capacity. Over the past two decades, much genetic modification work has been performed with the AAV capsid gene, resulting in a large number of variants with modified characteristics, rendering AAV a versatile vector for more efficient gene therapy applications for different genetic diseases.


Author(s):  
Guoxin Tan ◽  
Jiayang Li ◽  
Dandan Liu ◽  
Hao Pan ◽  
Renfang Zhu ◽  
...  

Nanomaterials ◽  
2021 ◽  
Vol 11 (4) ◽  
pp. 935
Author(s):  
Manas R. Biswal ◽  
Sofia Bhatia

Ocular gene therapy offers significant potential for preventing retinal dystrophy in patients with inherited retinal dystrophies (IRD). Adeno-associated virus (AAV) based gene transfer is the most common and successful gene delivery approach to the eye. These days, many studies are using non-viral nanoparticles (NPs) as an alternative therapeutic option because of their unique properties and biocompatibility. Here, we discuss the potential of carbon dots (CDs), a new type of nanocarrier for gene delivery to the retinal cells. The unique physicochemical properties of CDs (such as optical, electronic, and catalytic) make them suitable for biosensing, imaging, drug, and gene delivery applications. Efficient gene delivery to the retinal cells using CDs depends on various factors, such as photoluminescence, quantum yield, biocompatibility, size, and shape. In this review, we focused on different approaches used to synthesize CDs, classify CDs, various pathways for the intake of gene-loaded carbon nanoparticles inside the cell, and multiple studies that worked on transferring nucleic acid in the eye using CDs.


2021 ◽  
Vol 332 ◽  
pp. 210-224
Author(s):  
Gang Li ◽  
Shanshan He ◽  
Andreas G. Schätzlein ◽  
Robert M. Weiss ◽  
Darryl T. Martin ◽  
...  

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