Novel Lung Tropic Adeno-Associated Virus Capsids for Therapeutic Gene Delivery

2020 ◽  
Vol 31 (17-18) ◽  
pp. 996-1009
Author(s):  
Ana Carneiro ◽  
Hyuncheol Lee ◽  
Li Lin ◽  
Joost van Haasteren ◽  
David V. Schaffer
Keyword(s):  
Gene Delivery ◽  
Therapeutic Gene ◽  
Adeno Associated Virus ◽  
Virus Capsids

Recombinant Adeno-Associated Virus Type 8-Mediated Extensive Therapeutic Gene Delivery into Skeletal Muscle ofα-Sarcoglycan-Deficient Mice

2008 ◽  
Vol 19 (7) ◽  
pp. 719-730 ◽  
Author(s):  
Akiyo Nishiyama ◽  
Beryl Nyamekye Ampong ◽  
Sachiko Ohshima ◽  
Jin-Hong Shin ◽  
Hiroyuki Nakai ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Gene Delivery ◽  
Virus Type ◽  
Therapeutic Gene ◽  
Adeno Associated Virus ◽  
Deficient Mice

Recent advances in the analysis full/empty capsid ratio and genome integrity of adeno-associated virus (AAV) gene delivery vectors

2020 ◽  
Vol 20 ◽  
Author(s):  
L. Hajba ◽  
A. Guttman
Keyword(s):  
Gene Delivery ◽  
High Efficiency ◽  
Analytical Techniques ◽  
Viral Gene ◽  
Adeno Associated Virus ◽  
Gene Delivery Vectors ◽  
Empty Capsid ◽  
Purification Methods ◽  
Viral Gene Delivery

: Adeno-associated virus (AAV) is one of the most promising viral gene delivery vectors with long-term gene expression and disease correction featuring high efficiency and excellent safety in human clinical trials. During the production of AAV vectors,there are several quality control (QC)parameters that should be rigorously monitored to comply with clini-cal safety and efficacy. This review gives a short summary of the most frequently used AVV production and purification methods,focusing on the analytical techniques applied to determine the full/empty capsid ratio and the integrity of the encapsidated therapeutic DNA of the products.

scholarly journals Carbon Dot Nanoparticles: Exploring the Potential Use for Gene Delivery in Ophthalmic Diseases

Nanomaterials ◽  
2021 ◽  
Vol 11 (4) ◽  
pp. 935
Author(s):  
Manas R. Biswal ◽  
Sofia Bhatia
Keyword(s):  
Gene Delivery ◽  
Therapeutic Option ◽  
Retinal Dystrophy ◽  
Retinal Cells ◽  
Adeno Associated Virus ◽  
Inherited Retinal Dystrophies ◽  
Retinal Dystrophies ◽  
Efficient Gene ◽  
New Type ◽  
Viral Nanoparticles

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.

scholarly journals Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery

2017 ◽  
Vol 12 (1) ◽  
pp. 51-83 ◽  
Author(s):  
Chaitanya R. Joshi ◽  
Vinod Labhasetwar ◽  
Anuja Ghorpade
Keyword(s):  
Gene Delivery ◽  
Therapeutic Gene ◽  
The Past

scholarly journals Enhanced gene delivery in porcine vasculature tissue following incorporation of adeno-associated virus nanoparticles into porous silicon microparticles

2014 ◽  
Vol 194 ◽  
pp. 113-121 ◽  
Author(s):  
Kellie I. McConnell ◽  
Jessica Rhudy ◽  
Kenji Yokoi ◽  
Jianhua Gu ◽  
Aaron Mack ◽  
...  
Keyword(s):  
Porous Silicon ◽  
Gene Delivery ◽  
Adeno Associated Virus
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