Nanotechnology Now

Our NanoNews Digest Sponsors





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

Engineering a better 'Do: Purdue researchers are learning how August 4th, 2015

Proving nanoparticles in sunscreen products August 4th, 2015

Global Carbon Nanotubes Industry 2015: Acute Market Reports August 4th, 2015

Nanoparticles Give Antibacterial Properties to Machine-Woven Carpets August 4th, 2015

Possible Futures

Global Carbon Nanotubes Industry 2015: Acute Market Reports August 4th, 2015

Nanozirconia Market 2015 - Global Industry Survey, Analysis, Size, Share, Outlook and Forecast to 2020 July 31st, 2015

Self-Healing Nano Anti-rust Coatings Market 2015 - Global Industry Survey, Analysis, Size, Share, Outlook and Forecast to 2020 July 31st, 2015

Nano Spray Instrument Market 2015 - Global Industry Survey, Analysis, Size, Share, Outlook and Forecast to 2020 July 31st, 2015

Academic/Education

Pakistani Students Who Survived Terror Attack to Attend Weeklong “NanoDiscovery Institute” at SUNY Poly CNSE in Albany July 29th, 2015

Deben reports on the use of their CT500 in the X-ray microtomography laboratory at La Trobe University, Melbourne, Australia July 22nd, 2015

JPK reports on the use of SPM in the Messersmith Group at UC Berkeley looking at biologically inspired polymer adhesives. July 21st, 2015

Renishaw adds Raman analysis to Scanning Electron Microscopy at the University of Sydney, Australia July 9th, 2015

Nanomedicine

Nanoparticles used to breach mucus barrier in lungs: Proof-of-concept study conducted in mice a key step toward better treatments for lung diseases August 3rd, 2015

Promising Step Taken in Iran towards Treatment of Spinal Cord Injury August 3rd, 2015

Diagnosis of Salmonella Bacterium-Caused Food Poisoning by Biosensors August 3rd, 2015

Gold-diamond nanodevice for hyperlocalised cancer therapy: Gold nanorods can be used as remote controlled nanoheaters delivering the right amount of thermal treatment to cancer cells, thanks to diamond nanocrystals used as temperature sensors August 1st, 2015

Discoveries

Engineering a better 'Do: Purdue researchers are learning how August 4th, 2015

Proving nanoparticles in sunscreen products August 4th, 2015

Nanoparticles Give Antibacterial Properties to Machine-Woven Carpets August 4th, 2015

Quantum states in a nano-object manipulated using a mechanical system August 3rd, 2015

Announcements

Engineering a better 'Do: Purdue researchers are learning how August 4th, 2015

Proving nanoparticles in sunscreen products August 4th, 2015

Global Carbon Nanotubes Industry 2015: Acute Market Reports August 4th, 2015

Nanoparticles Give Antibacterial Properties to Machine-Woven Carpets August 4th, 2015

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

Heating and cooling with light leads to ultrafast DNA diagnostics July 31st, 2015

Springer and Tsinghua University Press present the second Nano Research Award: Paul Alivisatos of the University of California Berkeley receives the honor for outstanding contributions in nanoscience July 30th, 2015

European Technology Platform for Nanomedicine and ENATRANS European Consortium Launch the 2nd edition of the Nanomedicine Award: The Award to be presented at BIO-Europe conference in Munich, November 2015 July 30th, 2015

Publication on Atomic Force Microscopy based nanoscale IR Spectroscopy (AFM-IR) persists as a 2015 top downloaded paper July 29th, 2015

Nanobiotechnology

Heating and cooling with light leads to ultrafast DNA diagnostics July 31st, 2015

European Technology Platform for Nanomedicine and ENATRANS European Consortium Launch the 2nd edition of the Nanomedicine Award: The Award to be presented at BIO-Europe conference in Munich, November 2015 July 30th, 2015

New computer model could explain how simple molecules took first step toward life: Two Brookhaven researchers developed theoretical model to explain the origins of self-replicating molecules July 28th, 2015

Spintronics: Molecules stabilizing magnetism: Organic molecules fixing the magnetic orientation of a cobalt surface/ building block for a compact and low-cost storage technology/ publication in Nature Materials July 25th, 2015

Research partnerships

University of Puerto Rico announces August 11th as the launch date for their NASA mission to look for life in space – XEI reports August 3rd, 2015

Newly-Developed Polymers Control Size of Nanoparticles during Production Process July 30th, 2015

Meet the high-performance single-molecule diode: Major milestone in molecular electronics scored by Berkeley Lab and Columbia University team July 29th, 2015

Spintronics: Molecules stabilizing magnetism: Organic molecules fixing the magnetic orientation of a cobalt surface/ building block for a compact and low-cost storage technology/ publication in Nature Materials July 25th, 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