Printer Friendly
The Free Library
5,671,890 articles and books
Member login
User name  
Password 
 
Join us Forgot password?

AVI BioPharma Introduces ESPRIT With Initial Application in Muscular Dystrophy; New RNA Therapeutic Strategy Builds on NEUGENE Antisense Expertise.


PORTLAND, Ore. -- AVI BioPharma, Inc. (Nasdaq:AVII), today announced a new application of its proprietary NEUGENE(R) antisense antisense, DNA or RNA manipulated in a laboratory so that its components (nucleotides) form a complementary copy of normal, or "sense," messenger RNA (mRNA; see nucleic acid).  technology, called ESPRIT(TM) (Exon Exon

In split genes, a portion that is included in the ribonucleic acid (RNA) transcript of a gene and survives processing of the RNA in the cell nucleus to become part of a spliced messenger RNA (mRNA) or structural RNA in the cell cytoplasm.
 Skipping Pre-RNA Interference Technology). ESPRIT therapeutics are designed to either delete disease-causing genetic sequences or skip functional sequences to redesign proteins that are over-expressed or harmful in certain diseases.

"This is a new approach to solving genetic disorders and diseases caused by over-expressed or harmful genes," said Denis Denis, king of Portugal: see Diniz.  R. Burger, Ph.D., chief executive officer of AVI (Audio Video Interleaved) A Windows multimedia video format from Microsoft. It interleaves standard waveform audio and digital video frames (bitmaps) to provide reduced animation at 15 fps at 160x120x8 resolution. Audio is 11,025Hz, 8-bit samples. . "ESPRIT therapeutics allow for fine genetic surgery at the RNA RNA: see nucleic acid.
RNA
 in full ribonucleic acid

One of the two main types of nucleic acid (the other being DNA), which functions in cellular protein synthesis in all living cells and replaces DNA as the carrier of genetic
 processing level, providing a new and very potent tool for altering many disease mechanisms. In addition to genetic disorders such as muscular dystrophy, AVI is now applying the ESPRIT therapeutic approach to diseases with an immunologic component, such as diabetes and multiple sclerosis."

In normal genetic function, gene transcription produces a full-length pre-RNA that is then processed to a much shorter and functional messenger RNA. The mRNA is the template for creating a protein. During pre-RNA processing, packets of useful genetic information, called exons, are snipped out of the full-length RNA and spliced together to make the functional mRNA template. AVI's proprietary third-generation NEUGENE chemistry can be used to target splice-joining sites in the pre-RNA, thus forcing the cell machinery to skip over targeted exons, providing altered mRNA, which in turn produces altered proteins.

The first use of AVI's ESPRIT therapeutics was conducted in collaboration with Dr. Steve Wilton, associate professor and head of the Experimental Molecular Medicine Group at the Australian Neuromuscular Research Institute in Nedlands, Western Australia This article is about a suburb of Perth, Western Australia. For the local government area, see City of Nedlands.
Nedlands is a western suburb of Perth, Western Australia.
. Targeting the defective Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD)
The most severe form of muscular dystrophy, DMD usually affects young boys and causes progressive muscle weakness, usually beginning in the legs.
 (DMD (1) (Digital Micromirror Device) See DLP.

(2) (Digital Multi-layer Disk) See high-def DVD formats.
) dystrophin dys·tro·phin
n.
A structural protein found in small amounts in normal muscle but absent or present in abnormal amounts in individuals with muscular dystrophy.
 gene with an ESPRIT compound, Dr. Wilton was able to force the cell to snip out the disease-causing mutation in that region. Using this approach, a semifunctional dystrophin protein can be made from a DMD gene that would previously have only made a nonfunctional protein.

"Antisense oligomers can alter gene expression by snipping out the disease-causing mutation of a gene transcript during the splicing step of gene expression to convert DMD to the much less disabling Becker muscular dystrophy Beck·er muscular dystrophy
n.
A form of pseudohypertrophic muscular dystrophy that is less severe than Duchenne's muscular dystrophy, in which patients remain ambulatory and usually live until the third or fourth decade.
," Dr. Wilton said. "Morpholino antisense oligomers appear to be the most efficient chemistry approach for exon-skipping, as they exhibit low toxicity, have been administered systemically, persist for months and have already been used in human clinical trials."

Dr. Wilton has used the mdx mouse model of muscular dystrophy to show that the early stop signal in exon 23 can be efficiently skipped in the modified mRNA so that significant amounts of dystrophin are produced and correctly localized. The efficient delivery of some of these compounds generated very promising results with near-normal dystrophin being produced and persisting for months from a single treatment. AVI and Dr. Wilton have now extended these studies to other models of muscular dystrophy involved in the minor and major deletion hotspots in the human dystrophin gene. AVI and Dr. Wilton hope to take advantage of this existing data to commence clinical trials in the future.

Dr. Wilton will present research progress involving the ESPRIT approach for the treatment of muscular dystrophy at four upcoming international conferences.

Upcoming presentations highlighting AVI's collaborative work on muscular dystrophy with Dr. Wilton:

--Saturday, Oct. 1, 2005: "Exon skipping and the dystrophin gene: molecular by-pass surgery" at the 10th International Congress of the World Muscle Society at Iguassu Falls, Brazil

--Saturday, Oct. 22, 2005: "Exon Skipping and Duchenne Muscular Dystrophy: A Light at the End of the Tunnel" at the Parent Project UK Conference in London, England

--Thursday, Nov. 17, 2005: "Exon Skipping" in the Duchenne Muscular Dystrophy and Related Dystrophinopathies session of the Muscular Dystrophy Association The Muscular Dystrophy Association (MDA) is an organization founded in 1950 which combats muscular dystrophy and diseases of the nervous system and muscular system in general by funding research, providing medical and community services, and educating health professionals  Clinic Director's Meeting in Tucson, Ariz.

--Thursday, Dec. 8, 2005: "Duchenne Muscular Dystrophy and Exon Skipping" at the 41st Turkish National Neurology Congress in Istanbul, Turkey

About Muscular Dystrophy

Muscular dystrophy (MD) is the common name for several progressive hereditary diseases that cause muscles to weaken and degenerate. Each type has its own hereditary pattern, age of onset The age of onset is a medical term referring to the age at which an individual acquires, develops, or first experiences a condition or symptoms of a disease or disorder.

Diseases are often categorized by their ages of onset as congenital, infantile, juvenile, or adult.
 and rate of muscle loss. Different genetic alterations cause different types of muscular dystrophies. It is estimated that between 50,000 and 250,000 individuals are affected annually. This number seems to be growing each year due to improved technology for earlier diagnosis.

Within our gene makeup, there is an important muscle protein called dystrophin, which is one of the largest genes found to date. Dystrophin acts as the glue that holds muscles together by maintaining the structure of muscle cells. Dystrophin is also believed to carry signals between the inside and outside of muscle fibers. Without dystrophin, muscles are not able to operate properly and will eventually suffer progressive damage.

The dystrophin gene is carried on the X chromosome. Young men are therefore more susceptible to dystrophin damage because they have only one X chromosome. When a boy is diagnosed with Duchenne MD, his body is not able to produce any functional dystrophin. In Becker MD, a distorted but functional version of dystrophin is generated. In either disorder, muscle cells within the body gradually weaken and eventually die, without fully functional dystrophin.

About AVI BioPharma

AVI BioPharma develops therapeutic products for the treatment of life-threatening diseases using third-generation NEUGENE antisense drugs. AVI's lead NEUGENE antisense compound is designed to target cell proliferation disorders, including cardiovascular restenosis, cancer and polycystic kidney disease Polycystic Kidney Disease Definition

Polycystic kidney disease (PKD) is one of the most common of all life-threatening human genetic disorders.
. In addition to targeting specific genes in the body, AVI's antiviral program uses NEUGENE antisense compounds to combat disease by targeting single-stranded RNA viruses, including West Nile virus West Nile virus, microorganism and the infection resulting from it, which typically produces no symptoms or a flulike condition. The virus is a flavivirus and is related to a number of viruses that cause encephalitis. , hepatitis C virus
This page is for the virus. For the disease, see Hepatitis C.
The Hepatitis C virus (HCV) is a small (50 nm in size), enveloped, single-stranded, positive sense RNA virus in the family Flaviviridae.
, dengue virus and Ebola virus. AVI has introduced a NEUGENE-based exon-skipping technology called ESPRIT therapy. More information about AVI is available on the company's Web site at http://www.avibio.com.

"Safe Harbor" Statement under the Private Securities Litigation Reform Act The Private Securities Litigation Reform Act of 1995 (PSLRA) implemented several significant substantive changes affecting certain cases brought under the federal securities laws, including changes related to pleading, discovery, liability, class representation and awards fees and  of 1995: The statements that are not historical facts contained in this release are forward-looking statements that involve risks and uncertainties, including, but not limited to, the results of research and development efforts, the results of preclinical and clinical testing, the effect of regulation by the FDA FDA
abbr.
Food and Drug Administration


FDA,
n.pr See Food and Drug Administration.

FDA,
n.pr the abbreviation for the Food and Drug Administration.
 and other agencies, the impact of competitive products, product development, commercialization and technological difficulties, and other risks detailed in the company's Securities and Exchange Commission filings.
COPYRIGHT 2005 Business Wire
No portion of this article can be reproduced without the express written permission from the copyright holder.
Copyright 2005, Gale Group. All rights reserved. Gale Group is a Thomson Corporation Company.

 Reader Opinion

Title:

Comment:



 

Article Details
Printer friendly Cite/link Email Feedback
Publication:Business Wire
Geographic Code:1USA
Date:Sep 29, 2005
Words:1027
Previous Article:Leading Retailers and Industry Luminaries Discuss Holiday Optimization Strategies at WebTrends Second Retail Summit of 2005; Data-Driven Discipline,...
Next Article:Secure Computing's Sidewinder G2 Security Appliance an SMB Market Leader in Unified Threat Management.
Topics:



Related Articles
AVI BIOPHARMA INITIATES PHASE II CARDIOVASCULAR CLINICAL STUDY.
AVI BioPharma's NEUGENE Shows Success Against Deadly Ebola Virus; Results Published in Online Journal Public Library of Science (PLoS) Pathogens.
AVI BioPharma Presents Preclinical Results Preventing and Treating Diabetes Using Its ESPRIT Exon Skipping Technology.
AVI BioPharma Reports Second Quarter Financial Results; Conference Call Begins Today at 11:00 a.m. Eastern Time.
AVI BioPharma Reports Third Quarter Financial Results.
AVI BioPharma Announces Clinical Development Initiative for Muscular Dystrophy.
AVI BioPharma Completes Purchase of Building for GMP Manufacturing.
AVI BioPharma Reports Positive Pre-Clinical Influenza Data.
AVI BioPharma and Ercole Biotech Announce License and Drug Development Agreement for Duchenne Muscular Dystrophy and Beta Thalassemia.
AVI BioPharma Announces First Quarter Financial Results.

Terms of use | Copyright © 2009 Farlex, Inc. | Feedback | For webmasters | Submit articles