Multifunctional Chitosan Nanocarriers for Respiratory Disease Gene Therapy

2012 ◽  
pp. 191-216
Keyword(s):  
Gene Therapy ◽  
Respiratory Disease ◽  
Disease Gene

scholarly journals Human miRNA miR-675 inhibits DUX4 expression and may be exploited as a potential treatment for Facioscapulohumeral muscular dystrophy

2021 ◽  
Vol 12 (1) ◽  
Author(s):  
Nizar Y. Saad ◽  
Mustafa Al-Kharsan ◽  
Sara E. Garwick-Coppens ◽  
Gholamhossein Amini Chermahini ◽  
Madison A. Harper ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Muscular Dystrophy ◽  
Small Molecules ◽  
Skeletal Muscles ◽  
Disease Gene ◽  
Facioscapulohumeral Muscular Dystrophy ◽  
Potential Treatment ◽  
Cell Models ◽  
Powerful Approach ◽  
Dominant Disease

AbstractFacioscapulohumeral muscular dystrophy (FSHD) is a potentially devastating myopathy caused by de-repression of the DUX4 gene in skeletal muscles. Effective therapies will likely involve DUX4 inhibition. RNA interference (RNAi) is one powerful approach to inhibit DUX4, and we previously described a RNAi gene therapy to achieve DUX4 silencing in FSHD cells and mice using engineered microRNAs. Here we report a strategy to direct RNAi against DUX4 using the natural microRNA miR-675, which is derived from the lncRNA H19. Human miR-675 inhibits DUX4 expression and associated outcomes in FSHD cell models. In addition, miR-675 delivery using gene therapy protects muscles from DUX4-associated death in mice. Finally, we show that three known miR-675-upregulating small molecules inhibit DUX4 and DUX4-activated FSHD biomarkers in FSHD patient-derived myotubes. To our knowledge, this is the first study demonstrating the use of small molecules to suppress a dominant disease gene using an RNAi mechanism.

Novel chimeric p16 and p27 molecules with increased antiproliferative activity for vascular disease gene therapy

2000 ◽  
Vol 78 (8) ◽  
pp. 451-459 ◽  
Author(s):  
Lou Lamphere ◽  
Lisa Tsui ◽  
Scott Wick ◽  
Takayuki Nakano ◽  
Lydia Kilinski ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Vascular Disease ◽  
Antiproliferative Activity ◽  
Disease Gene

scholarly journals Fiber-Modified Adenovirus for Central Nervous System Parkinson’s Disease Gene Therapy

Viruses ◽  
2014 ◽  
Vol 6 (8) ◽  
pp. 3293-3310 ◽  
Author(s):  
Travis Lewis ◽  
Joel Glasgow ◽  
Ashley Harms ◽  
David Standaert ◽  
David Curiel
Keyword(s):  
Parkinson’S Disease ◽  
Gene Therapy ◽  
Central Nervous System ◽  
Parkinson's Disease ◽  
Nervous System ◽  
Disease Gene

scholarly journals Pompe disease gene therapy

2011 ◽  
Vol 20 (R1) ◽  
pp. R61-R68 ◽  
Author(s):  
B. J. Byrne ◽  
D. J. Falk ◽  
C. A. Pacak ◽  
S. Nayak ◽  
R. W. Herzog ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Pompe Disease ◽  
Disease Gene

scholarly journals The Use of Viral Vectors in Gene Transfer Therapy

2016 ◽  
Vol 8 (03) ◽  
Author(s):  
A. Dziaková ◽  
A. Valenčáková ◽  
E. Hatalová ◽  
J. Kalinová
Keyword(s):  
Gene Therapy ◽  
Plasmid Dna ◽  
Clinical Studies ◽  
Viral Vectors ◽  
Disease Gene ◽  
Major Gene ◽  
Genetic Material ◽  
The Body ◽  
Disease Genes ◽  
Wide Range

Gene therapy is strategy based on using genes as pharmaceuticals. Gene therapy is a treatment that involves altering the genes inside body's cells to stop disease. Genes contain DNA- the code controlling body form and function. Genes that do not work properly can cause disease. Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve the ability of the body to fight disease. Gene therapy holds promise for treating a wide range of diseases, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Various types of genetic material are used in gene therapy; double-stranded DNA (dsDNA), single-stranded DNA (ssDNA), plasmid DNA and antisense oligodeoxynucleotides (ASON). The success of gene therapy depends on assuring the entrance of the therapeutic gene to targeted cells without any form of biodegradation. Commonly used vectors in gene therapy are: adenoviruses (400 clinical studies; 23.8%), retroviruses (344 clinical studies; 20.5%), unenveloped/plasmid DNA (304 clinical studies, 17.7%), adenoassociated viruses (75 clinical studies; 4.5%) and others. In this paper, we have reviewed the major gene delivery vectors and recent improvements made in their design meant to overcome the issues that commonly arise with the use of gene therapy vectors.

Sign in / Sign up

Export Citation Format

Share Document