Nanotechnology Now







Heifer International

Wikipedia Affiliate Button


DHgate

Home > Press > Breakthrough Gene Therapy Prevents Retinal Degeneration

Image on left shows damage (pink) to the retina. Image on right show that POD GDNF nanoparticles protected the retina from damage. (Image courtesy of Rajendra Kumar-Singh, Tufts University School of Medicine)
Image on left shows damage (pink) to the retina. Image on right show that POD GDNF nanoparticles protected the retina from damage. (Image courtesy of Rajendra Kumar-Singh, Tufts University School of Medicine)

Abstract:
In one of only two studies of its kind, a study from researchers at Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts demonstrates that non-viral gene therapy can delay the onset of some forms of eye disease and preserve vision. The team developed nanoparticles to deliver therapeutic genes to the retina and found that treated mice temporarily retained more eyesight than controls. The study, published online in advance of print in Molecular Therapy, brings researchers closer to a non-viral gene therapy treatment for degenerative eye disorders.

Breakthrough Gene Therapy Prevents Retinal Degeneration

Boston, MA | Posted on August 16th, 2010

"Our work shows that it is possible to attain therapeutic results using non-viral gene delivery methods, specifically, nanoparticles. Nanoparticles, which are small enough to penetrate cells and stable enough to protect DNA, are capable of preventing retinal cell death and preserving vision," said senior author Rajendra Kumar-Singh, PhD, associate professor of ophthalmology at Tufts University School of Medicine (TUSM) and member of the genetics; neuroscience; and cell, molecular, and developmental biology program faculties at the Sackler School of Graduate Biomedical Sciences at Tufts.

"The most common approach to gene therapy involves using a virus to deliver DNA to cells. While viruses are very efficient carriers, they can prompt immune responses that may lead to inflammation, cancer, or even death. Non-viral methods offer a safer alternative, but until now, efficiency has been a significant barrier," said Kumar-Singh.

In a model simulating the progression of human retinal degeneration, the researchers treated mice with nanoparticles carrying a gene for GDNF (Glial Cell Line-Derived Neurotrophic Factor), a protein known to protect the photoreceptor cells in the eye. Retinas treated with the GDNF-carrying nanoparticles showed significantly less photoreceptor cell death than controls. Preservation of these cells resulted in significantly better eyesight in the treatment group seven days after treatment, compared to controls.

The protection conferred by the GDNF-carrying nanoparticles was temporary, as tests fourteen days after treatment showed no difference in eyesight between treated mice and controls.

"The next step in this research is to prolong this protection by adding elements to the DNA that permit its retention in the cell. Bringing forth a more potent and enduring result will move us closer to clinical application of non-viral gene therapy," said Kumar-Singh.

AMD, which results in a loss of sharp, central vision, is the number one cause of visual impairment among Americans age 60 and older. Retinitis pigmentosa, an inherited condition characterized by night blindness and loss of peripheral vision, affects approximately 1 in 4,000 individuals in the United States.

Additional authors on the study are first author Sarah Parker Read, an MD/PhD candidate at TUSM and Sackler and member of Kumar-Singh's lab, and Siobhan Cashman, PhD, research assistant professor in the department of ophthalmology at TUSM and member of Kumar-Singh's lab.

In a previous study, this same team of researchers developed the gene delivery method used in this research. The researchers showed that a peptide called PEG-POD, which compacts DNA into nanoparticles, delivers genes to the retina more efficiently than other non-viral carriers.

This study was supported by grants from The Ellison Foundation; the National Eye Institute, part of the National Institutes of Health; the Virginia B. Smith Trust; and grants to the Department of Ophthalmology at Tufts University from the Lions Eye Foundation and Research to Prevent Blindness. Sarah Parker Read is part of the Sackler/TUSM Medical Scientist Training Program, which is funded by the National Institute of General Medical Sciences, part of the National Institutes of Health.

Read SP, Cashman SM, Kumar-Singh R. Molecular Therapy. "POD Nanoparticles Expressing GDNF Provide Structural and Functional Rescue of Light-Induced Retinal Degeneration in an Adult Mouse." Published online August 10, 2010, doi: 10.1038/mt.2010.167

####

About Tufts University School of Medicine
Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts University are international leaders in innovative medical education and advanced research. The School of Medicine and the Sackler School are renowned for excellence in education in general medicine, biomedical sciences, special combined degree programs in business, health management, public health, bioengineering and international relations, as well as basic and clinical research at the cellular and molecular level. Ranked among the top in the nation, the School of Medicine is affiliated with six major teaching hospitals and more than 30 health care facilities. Tufts University School of Medicine and the Sackler School undertake research that is consistently rated among the highest in the nation for its effect on the advancement of medical science.

For more information, please click here

Contacts:
Siobhan Gallagher
617-636-6586

Copyright © Tufts University School of Medicine

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

MIG Takes a Roll-Up-Your-Sleeves Approach with Revamped MEMS/Sensors Technical Event -- MIG welcomes technologists to MEMS Technical Congress, emphasizes working groups and breakout sessions on emerging MEMS & sensors, tech transfer and integration March 6th, 2015

Phenom-World announces the Phenom XL, world’s fastest desktop SEM to handle large samples March 6th, 2015

Air Bearing Stage / Systems Introduced by PI at Photonics West March 6th, 2015

Consistent Scalable Functionalised Graphene Capacity March 5th, 2015

Possible Futures

European roadmap for graphene science and technology published February 25th, 2015

Quantum research past, present and future for discussion at AAAS February 16th, 2015

World’s first compact rotary 3D printer-cum-scanner unveiled at AAAS by NTU Singapore start-up: With production funded by crowdsourcing, the first unit will be delivered to the United States in March February 16th, 2015

Nanotechnology Electric Vehicle (EV) Market Analysis Report 2015: According to Radiant Insights, Inc February 13th, 2015

Academic/Education

Get ready for NanoDays! March 5th, 2015

NanoTecNexus Launches New App for Learning About Nanotechnology—STEM Education Project Spearheaded by Interns February 26th, 2015

SUNY Poly CNSE Researchers and Corporate Partners to Present Forty Papers at Globally Recognized Lithography Conference: SUNY Poly CNSE Research Group Awarded Both ‘Best Research Paper’ and ‘Best Research Poster’ at SPIE Advanced Lithography 2015 forum February 25th, 2015

KIT Increases Commitment in Asia: DAAD Funds Two New Projects: Strategic Partnerships with Chinese Universities and Communi-cation Technologies Network February 22nd, 2015

Nanomedicine

Patent for the Novel Cancer Therapies – Ceramide Nanoliposomes March 4th, 2015

Arrowhead to Present at 2015 Barclays Global Healthcare Conference March 4th, 2015

Democratizing synthetic biology: New method makes research cheaper, faster, and more accessible March 3rd, 2015

Pens filled with high-tech inks for do-it-yourself sensors March 3rd, 2015

Discoveries

Enhanced Graphene Components for Next Generation Racing Yacht March 5th, 2015

American Chemical Society Presidential Symposia: nanoscience, international chemistry March 5th, 2015

Strength in numbers: Researchers develop the first-ever quantum device that detects and corrects its own errors March 4th, 2015

New research could lead to more efficient electrical energy storage March 4th, 2015

Announcements

MIG Takes a Roll-Up-Your-Sleeves Approach with Revamped MEMS/Sensors Technical Event -- MIG welcomes technologists to MEMS Technical Congress, emphasizes working groups and breakout sessions on emerging MEMS & sensors, tech transfer and integration March 6th, 2015

Phenom-World announces the Phenom XL, world’s fastest desktop SEM to handle large samples March 6th, 2015

Air Bearing Stage / Systems Introduced by PI at Photonics West March 6th, 2015

Get ready for NanoDays! March 5th, 2015

Grants/Awards/Scholarships/Gifts/Contests/Honors/Records

Anousheh Ansari Wins the National Space Society's Space Pioneer Award for "Service to the Space Community" March 5th, 2015

Enhanced Graphene Components for Next Generation Racing Yacht March 5th, 2015

Rice's Stephan Link honored for nanoscience research: The Welch Foundation honors ‘rising star’ with $100,000 Hackerman Award February 26th, 2015

Cutting-edge technology optimizes cancer therapy with nanomedicine drug combinations: UCLA bioengineers develop platform that offers personalized approach to treatment February 24th, 2015

Nanobiotechnology

Experiment and theory unite at last in debate over microbial nanowires: New model and experiments settle debate over metallic-like conductivity of microbial nanowires in bacterium March 4th, 2015

Untangling DNA with a droplet of water, a pipet and a polymer: With the 'rolling droplet technique,' a DNA-injected water droplet rolls like a ball over a platelet, sticking the DNA to the plate surface February 27th, 2015

Bacteria network for food: Bacteria connect to each other and exchange nutrients February 23rd, 2015

Building tailor-made DNA nanotubes step by step: New, block-by-block assembly method could pave way for applications in opto-electronics, drug delivery February 23rd, 2015

Research partnerships

French Institutes IRT Nanoelec and CMP Team up to Offer World’s First Service for Post-process 3D Technologies on Multi-Project-Wafer March 5th, 2015

New research could lead to more efficient electrical energy storage March 4th, 2015

Cambrios and Heraeus Jointly Create New, High-Conductivity Transparent Conductors: Two Companies' Combined Products Dramatically Extend Flexible Substrate Capabilities for Next-Generation Mass-Market Technology Products March 3rd, 2015

The taming of magnetic vortices: Unified theory for skyrmion-materials March 3rd, 2015

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 1999-2015 7th Wave, Inc. All Rights Reserved PRIVACY POLICY :: CONTACT US :: STATS :: SITE MAP :: ADVERTISE