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

Metal oxide sandwiches: New option to manipulate properties of interfaces February 8th, 2016

Canadian physicists discover new properties of superconductivity February 8th, 2016

Leading bugs to the death chamber: A kinder face of cholesterol February 8th, 2016

From allergens to anodes: Pollen derived battery electrodes February 8th, 2016

Possible Futures

From allergens to anodes: Pollen derived battery electrodes February 8th, 2016

Host-guest nanowires for efficient water splitting and solar energy storage February 7th, 2016

Graphene is strong, but is it tough? Berkeley Lab scientists find that polycrystalline graphene is not very resistant to fracture February 7th, 2016

Scientists take key step toward custom-made nanoscale chemical factories: Berkeley Lab researchers part of team that creates new function in tiny protein shell structures February 6th, 2016

Academic/Education

COD Grad Begins Postdoctoral Fellow at Harvard University: Marsela Jorgolli's Passion for Physics Has Led to a Decade of Academic Research That Continues at Harvard University as a Postdoctoral Fellow February 2nd, 2016

Heriot-Watt's Institute of Photonics & Quantum Sciences uses the Deben Microtest 2 kN tensile stage to characterise ceramics and engineering plastics January 21st, 2016

Multiple uses for the JPK NanoWizard AFM system in the Smart Interfaces in Environmental Nanotechnology Group at the University of Illinois at Urbana-Champaign January 20th, 2016

BioSolar Extends Research Agreement With UCSB for Next Phase of Its Super Battery Technology: Development Effort to Continue Under the Supervision of Nobel Laureate, Dr. Alan Heeger January 13th, 2016

Nanomedicine

Leading bugs to the death chamber: A kinder face of cholesterol February 8th, 2016

UTHealth research looks at nanotechnology to help prevent preterm birth February 7th, 2016

Scientists take key step toward custom-made nanoscale chemical factories: Berkeley Lab researchers part of team that creates new function in tiny protein shell structures February 6th, 2016

Hepatitis virus-like particles as potential cancer treatment February 5th, 2016

Discoveries

Metal oxide sandwiches: New option to manipulate properties of interfaces February 8th, 2016

Canadian physicists discover new properties of superconductivity February 8th, 2016

Leading bugs to the death chamber: A kinder face of cholesterol February 8th, 2016

The iron stepping stones to better wearable tech without semiconductors February 8th, 2016

Announcements

Metal oxide sandwiches: New option to manipulate properties of interfaces February 8th, 2016

Canadian physicists discover new properties of superconductivity February 8th, 2016

Leading bugs to the death chamber: A kinder face of cholesterol February 8th, 2016

From allergens to anodes: Pollen derived battery electrodes February 8th, 2016

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

UTHealth research looks at nanotechnology to help prevent preterm birth February 7th, 2016

Graphene is strong, but is it tough? Berkeley Lab scientists find that polycrystalline graphene is not very resistant to fracture February 7th, 2016

Hepatitis virus-like particles as potential cancer treatment February 5th, 2016

Identifying Commercial Success Stories from the National Nanotechnology Initiative: National Nanotechnology Coordination Office and White House Office of Science and Technology Policy Issue a Request for Information on NNI-Supported Success Stories February 2nd, 2016

Nanobiotechnology

Leading bugs to the death chamber: A kinder face of cholesterol February 8th, 2016

UTHealth research looks at nanotechnology to help prevent preterm birth February 7th, 2016

Scientists take key step toward custom-made nanoscale chemical factories: Berkeley Lab researchers part of team that creates new function in tiny protein shell structures February 6th, 2016

Hepatitis virus-like particles as potential cancer treatment February 5th, 2016

Research partnerships

Scientists take key step toward custom-made nanoscale chemical factories: Berkeley Lab researchers part of team that creates new function in tiny protein shell structures February 6th, 2016

Polar vortices observed in ferroelectric: New state of matter holds promise for ultracompact data storage and processing February 4th, 2016

Spin dynamics in an atomically thin semi-conductor February 1st, 2016

Graphene shown to safely interact with neurons in the brain January 31st, 2016

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







Car Brands
Buy website traffic