Nanotechnology Now

Our NanoNews Digest Sponsors



Heifer International

Wikipedia Affiliate Button


android tablet pc

Home > Press > GeneSegues and Collaborators at University of Minnesota Publish New Research Showing Targeted Nanocapsule Delivery of Plasmid DNA for Long Term Treatment of Hemophilia in Mice

Abstract:
GeneSegues, Inc., a biopharmaceutical company developing and commercializing novel nanocapsule-based drug delivery technology, announced today a publication in the Journal of Clinical Investigation demonstrating that systemic administration of plasmid DNA in liver-targeted, non-viral nanocapsules resulted in complete and long term phenotypic correction of hemophilia A in a mouse model.

GeneSegues and Collaborators at University of Minnesota Publish New Research Showing Targeted Nanocapsule Delivery of Plasmid DNA for Long Term Treatment of Hemophilia in Mice

Chaska, MN | Posted on June 9th, 2009

The work is the result of collaborative studies between GeneSegues and the laboratory of Clifford Steer, M.D., Professor of Medicine, University of Minnesota, and was supported by grants from the National Heart, Lung, and Blood Institute, the National Institute of Diabetes and Digestive and Kidney Diseases, and the Department of Defense.

"Hemophilia A is an X-linked congenital bleeding disorder caused by deficiency of coagulation Factor VIII, affecting more than one-half million males worldwide," said lead author Betsy Kren, Ph.D., Assistant Professor of Medicine, University of Minnesota. "Cell-specific delivery of DNA plasmid to the endogenous production site for Factor VIII, to restore Factor VIII levels for an extended period without the development of anti-Factor VIII antibodies, could contribute significantly to a more effective treatment of hemophilia A. To date, no non-viral approach has been identified that is capable of achieving such results. We believe these findings may represent a major advance in the field of gene therapy, and are very encouraged by the results published today with GeneSegues."

The work described in the publication (Kren et al., Journal of Clinical Investigation,
advance online publication 8 June 2009; doi: 10.1172/JCI34332; available at www.jci.org/articles/view/34332), uses the transgene plasmid ‘Sleeping Beauty', expressing B-domain deleted canine Factor VIII and encapsulated in sub-50 nanometer capsules with a hyaluronan ligand shell targeting specifically to liver sinusoidal endothelial cells, to restore Factor VIII levels in Factor VIII knockout mice. The animals were monitored for clotting time by measuring activated partial thromboplastin times (aPTT) over a period of 50 weeks. Single-dose intravenous delivery of the encapsulated plasmid resulted in improvement of aPTT of the treated mice to levels statistically equivalent to wild-type controls through the 50-week study period, while all untreated controls died before the end of the study. Importantly, results showed that the encapsulated plasmid did not activate anti-Factor VIII antibodies.

"Our collaboration with Drs. Steer and Kren and their colleagues has been very productive, and we are pleased to have the opportunity to work with a leading group of researchers in this area," said co-author Gretchen Unger, Ph.D., President of GeneSegues. "We are very encouraged by the results of this hemophilia work, as well as complementary work with reporter genes showing that specific uptake by different liver cell-types corresponds with the applicable capsule targeting ligand. New approaches to gene therapy using nonviral delivery systems may pave the way for novel disease-modifying medicines for hemophilia as well as other diseases of the liver."

####

About GeneSegues
GeneSegues is a privately-held biopharmaceutical company focused on developing and commercializing novel nanocapsule-based drug delivery technology for DNA and RNA-based therapeutics. The company’s sub-50 nanometer (“s50”) capsule technology is unique in the capability to co-opt nondegradative pathways into the target cell, avoiding intracellular drug degradation that has thus far thwarted commercialization of DNA/RNA-based therapeutics. GeneSegues has expertise in working with all types of nucleic acid-based drugs including plasmids and single and double-strand RNAi, in tissue culture and animal models. The company has established collaborative relationships with larger drug developers, whereby s50 technology is being used to deliver nucleic acid-based therapeutics to a broad range of targets, via systemic as well as topical administration.

For more information, please click here

Contacts:
Gretchen M. Unger
President

952-443-3798

Copyright © GeneSegues

If you have a comment, please Contact us.

Issuers of news releases, not 7th Wave, Inc. or Nanotechnology Now, are solely responsible for the accuracy of the content.

Bookmark:
Delicious Digg Newsvine Google Yahoo Reddit Magnoliacom Furl Facebook

Related News Press

News and information

Scientists reveal breakthrough in optical fiber communications December 21st, 2014

Atom-thick CCD could capture images: Rice University scientists develop two-dimensional, light-sensitive material December 20th, 2014

Oregon researchers glimpse pathway of sunlight to electricity: Collaboration with Lund University uses modified UO spectroscopy equipment to study 'maze' of connections in photoactive quantum dots December 19th, 2014

Instant-start computers possible with new breakthrough December 19th, 2014

Possible Futures

A novel method for identifying the body’s ‘noisiest’ networks November 19th, 2014

Researchers discern the shapes of high-order Brownian motions November 17th, 2014

VDMA Electronics Production Equipment: Growth track for 2014 and 2015 confirmed: Business climate survey shows robust industry sector November 14th, 2014

Open Materials Development Will Be Key for HP's Success in 3D Printing: HP can make a big splash in 3D printing, but it needs to shore up technology claims and avoid the temptation of the razor/razor blade business model in order to flourish November 11th, 2014

Nanomedicine

Creation of 'Rocker' protein opens way for new smart molecules in medicine, other fields December 18th, 2014

Iranian Researchers Produce Electrical Pieces Usable in Human Body December 18th, 2014

Unraveling the light of fireflies December 17th, 2014

First Home-Made Edible Herbal Nanodrug Presented to Pharmacies across Iran December 17th, 2014

Announcements

Scientists reveal breakthrough in optical fiber communications December 21st, 2014

Atom-thick CCD could capture images: Rice University scientists develop two-dimensional, light-sensitive material December 20th, 2014

Oregon researchers glimpse pathway of sunlight to electricity: Collaboration with Lund University uses modified UO spectroscopy equipment to study 'maze' of connections in photoactive quantum dots December 19th, 2014

Instant-start computers possible with new breakthrough December 19th, 2014

Nanobiotechnology

Scientists trace nanoparticles from plants to caterpillars: Rice University study examines how nanoparticles behave in food chain December 16th, 2014

FEI and Oregon Health & Science University Install a Complete Correlative Microscopy Workflow in Newly Built Collaborative Science Facility December 16th, 2014

UCLA engineers first to detect and measure individual DNA molecules using smartphone microscope December 15th, 2014

Biomimetic dew harvesters: Understanding how a desert beetle harvests water from dew could improve drinking water collection in dew condensers December 8th, 2014

NanoNews-Digest
The latest news from around the world, FREE




  Premium Products
NanoNews-Custom
Only the news you want to read!
 Learn More
NanoTech-Transfer
University Technology Transfer & Patents
 Learn More
NanoStrategies
Full-service, expert consulting
 Learn More










ASP
Nanotechnology Now Featured Books




NNN

The Hunger Project







© Copyright 1999-2014 7th Wave, Inc. All Rights Reserved PRIVACY POLICY :: CONTACT US :: STATS :: SITE MAP :: ADVERTISE