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https://www.ionispharma.com/ionis-innovation/antisense-technology/
Antisense therapies are designed to seek out, bind to and destroy a mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased. Antisense therapies can also treat diseases caused by too little protein by increasing the production of the protein, thereby restoring the protein to normal levels.
https://ir.ionispharma.com/news-releases/news-release-details/isis-pharmaceuticals-demonstrates-potential-single-stranded-rna
Aug 30, 2012 · Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners. Isis' broad pipeline consists of 25 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and …
https://www.sciencedirect.com/science/article/pii/S1474442213700619
This trial is the first clinical study of intrathecal delivery of an antisense oligonucleotide. ISIS 333611 was well tolerated when administered as an intrathecal infusion. Antisense oligonucleotides delivered to the CNS might be a feasible treatment for neurological disorders.Author: Timothy M. Miller, Alan Pestronk, William David, Jeffrey Rothstein, Ericka P. Simpson, Stanley H. Ap...
https://www.ncbi.nlm.nih.gov/pubmed/23541756
ISIS 333611 was well tolerated when administered as an intrathecal infusion. Antisense oligonucleotides delivered to the CNS might be a feasible treatment for neurological disorders. FUNDING: The ALS Association, Muscular Dystrophy Association, Isis Pharmaceuticals.Author: Timothy M. Miller, Alan Pestronk, William David, Jeffrey Rothstein, Ericka P. Simpson, Stanley H. Ap...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4117763/
Sep 01, 2014 · Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in miceAuthor: Thazha P. Prakash, Mark J. Graham, Jinghua Yu, Rick Carty, Audrey Low, Alfred Chappell, Karsten Schm...
https://www.ionispharma.com/ionis-innovation/pipeline/
IONIS-HBV Rx is an antisense drug designed to reduce the production of viral proteins associated with hepatitis B virus (HBV) infection and replication, including hepatitis B surface antigen, which is present in both acute and chronic infections and is associated with a poor prognosis in patients with chronic HBV infection. HBV infection is a serious health problem that can lead to significant and potentially fatal …
https://cms.psav.com/efb7b65/public/download_uploaded_media/pdf/64
Isis Pharmaceuticals, Inc. Carlsbad, CA USA PK and PD Properties of Antisense Oligonucleotides: Bridging Nonclinical to Clinical . Antisense Mechanism of Action ... In Advanced Delivery and Therapeutic Applications of RNAi, First Edition. Edited by Kun Cheng and Ram I. Mahato. 2013.
https://mct.aacrjournals.org/content/1/5/347
Mar 01, 2002 · The use of vectors in antisense drug delivery in vivo remains, at this point, a somewhat open question. In contrast to in vitro studies, all of the clinical trials with antisense oligonucleotides are carried out with naked oligonucleotides (51, 104). A delivery vehicle does not appear to be needed as endosomal/lysosomal sequestration, and lack of nuclear localization does not appear to be a problem.Author: Nathalie Dias, C. A. Stein
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