Topical gene silencing by iontophoretic delivery of an antisense oligonucleotide–dendrimer nanocomplex: the proof of concept in a skin cancer mouse model

Nanoscale ◽  
2015 ◽  
Vol 7 (9) ◽  
pp. 3903-3914 ◽  
Author(s):  
Venkata Vamsi K. Venuganti, ◽  
Manju Saraswathy ◽  
Chandradhar Dwivedi ◽  
Radhey S. Kaushik ◽  
Omathanu P. Perumal
Keyword(s):  
Skin Cancer ◽  
Mouse Model ◽  
Gene Silencing ◽  
Antisense Oligonucleotide ◽  
Skin Diseases ◽  
Proof Of Concept ◽  
Iontophoretic Delivery

Topical iontophoretic delivery of a dendrimer–antisense oligonucleotide nanocomplex demonstrates the potential for developing gene silencing therapy for skin diseases.

Dendrosome mediated topical gene silencing by PLK-1 specific siRNA: implication in treatment of skin cancer in mouse model

RSC Advances ◽  
2016 ◽  
Vol 6 (8) ◽  
pp. 6843-6857
Author(s):  
Mohd. Asif Sherwani ◽  
Saba Tufail ◽  
Aijaz Ahmed Khan ◽  
Mohammad Owais
Keyword(s):  
Skin Cancer ◽  
Mouse Model ◽  
Gene Silencing ◽  
Topical Application ◽  
Anti Cancer ◽  
Skin Papillomas

Topical application of PLK-1 siRNA bearing dendrosomes on DMBA induced skin papillomas in mice exhibit potent anti-cancer effect. The treatment leads to reduced number and sizes of papillomas.

scholarly journals Antisense oligonucleotide therapy for KCNT1 encephalopathy

2020 ◽  
Author(s):  
Lisseth Estefania Burbano ◽  
Melody Li ◽  
Nikola Jancovski ◽  
Paymaan Jafar-Nejad ◽  
Kay Richards ◽  
...  
Keyword(s):  
Gene Silencing ◽  
Antisense Oligonucleotide ◽  
Therapeutic Approach ◽  
Bolus Injection ◽  
De Novo ◽  
Proof Of Concept ◽  
Epileptic Encephalopathies ◽  
Behavioral Abnormalities ◽  
Promising Therapeutic Approach ◽  
Antisense Oligonucleotide Therapy

ABSTRACTDevelopmental and epileptic encephalopathies (DEE) are characterized by pharmacoresistant seizures with concomitant intellectual disability. Epilepsy of infancy with migrating focal seizures (EIMFS) is one of the most severe of these syndromes. De novo mutations in ion channels, including gain-of-function variants in KCNT1, have been found to play a major role in the etiology of EIMFS. Here, we test a potential precision therapeutic approach in KCNT1-associated DEE using a gene silencing antisense oligonucleotide (ASO) approach. The homozygous p.P924L (L/L) mouse model recapitulates the frequent, debilitating seizures and developmental compromise that are seen in patients. After a single intracerebroventricular bolus injection of a Kcnt1 gapmer ASO in symptomatic mice at postnatal day 40, seizure frequency was significantly reduced, behavioral abnormalities improved, and overall survival was extended compared to mice treated with a control ASO (non-hybridizing sequence). ASO administration at neonatal age was also well-tolerated and effective in controlling seizures and extending the lifespan of treated animals. The data presented here provides a proof of concept for ASO-based gene silencing as a promising therapeutic approach in KCNT1-associated epilepsies.

Gene silencing with siRNA targeting E6/E7 as a therapeutic intervention in a mouse model of cervical cancer

2008 ◽  
Vol 111 (2) ◽  
pp. 356-364 ◽  
Author(s):  
Amy L. Jonson ◽  
Lisa M. Rogers ◽  
Sundaram Ramakrishnan ◽  
Levi S. Downs
Keyword(s):  
Cervical Cancer ◽  
Mouse Model ◽  
Gene Silencing ◽  
Therapeutic Intervention

scholarly journals Tumor Survivin Is Downregulated by the Antisense Oligonucleotide LY2181308: A Proof-of-Concept, First-in-Human Dose Study

2010 ◽  
Vol 16 (24) ◽  
pp. 6150-6158 ◽  
Author(s):  
Denis C. Talbot ◽  
Malcolm Ranson ◽  
Joanna Davies ◽  
Michael Lahn ◽  
Sophie Callies ◽  
...  
Keyword(s):  
Antisense Oligonucleotide ◽  
Proof Of Concept ◽  
Human Dose ◽  
Dose Study

scholarly journals MicroRNA-203 represses selection and expansion of oncogenic Hras transformed tumor initiating cells

eLife ◽  
2015 ◽  
Vol 4 ◽  
Author(s):  
Kent Riemondy ◽  
Xiao-jing Wang ◽  
Enrique C Torchia ◽  
Dennis R Roop ◽  
Rui Yi
Keyword(s):  
Cell Division ◽  
Skin Cancer ◽  
Mouse Model ◽  
Mouse Models ◽  
Knockout Mouse ◽  
Selection Process ◽  
Tumor Initiating Cells ◽  
Knockout Mouse Model ◽  
Active Selection ◽  
The Impact

In many mouse models of skin cancer, only a few tumors typically form even though many cells competent for tumorigenesis receive the same oncogenic stimuli. These observations suggest an active selection process for tumor-initiating cells. Here, we use quantitative mRNA- and miR-Seq to determine the impact of HrasG12V on the transcriptome of keratinocytes. We discover that microRNA-203 is downregulated by HrasG12V. Using a knockout mouse model, we demonstrate that loss of microRNA-203 promotes selection and expansion of tumor-initiating cells. Conversely, restoration of microRNA-203 using an inducible model potently inhibits proliferation of these cells. We comprehensively identify microRNA-203 targets required for Hras-initiated tumorigenesis. These targets include critical regulators of the Ras pathway and essential genes required for cell division. This study establishes a role for the loss of microRNA-203 in promoting selection and expansion of Hras mutated cells and identifies a mechanism through which microRNA-203 antagonizes Hras-mediated tumorigenesis.

scholarly journals Periostin antisense oligonucleotide prevents hepatic steatosis and fibrosis in a mouse model of non‐alcoholic steatohepatitis

2020 ◽  
Vol 35 (12) ◽  
pp. 2140-2150 ◽  
Author(s):  
Tomoki Kobayashi ◽  
Keishi Kanno ◽  
Phuong Thao Nguyen ◽  
Akiko Sugiyama ◽  
Akihiro Kawahara ◽  
...  
Keyword(s):  
Mouse Model ◽  
Hepatic Steatosis ◽  
Antisense Oligonucleotide ◽  
Alcoholic Steatohepatitis
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