Nanotechnology Now

Our NanoNews Digest Sponsors
Heifer International



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

The National Space Society Congratulates Blue Origin and Jeff Bezos for the Spectacular First Crewed Flight of the New Shepard: Well-Tested Suborbital Tourist Rocket Soars to 63 Miles; Opens New Frontiers July 21st, 2021

Unconventional superconductor acts the part of a promising quantum computing platform: If it looks like a duck, swims like a duck and quacks like a duck, then it probably is a duck. July 16th, 2021

Unlocking efficient light-energy conversion with stable coordination nanosheets: Scientists design a high-performance, self-powered, UV photodetector using 2D nanosheets that show record photocurrent stability under air exposure July 16th, 2021

The virus trap: Hollow nano-objects made of DNA could trap viruses and render them harmless July 16th, 2021

Possible Futures

The National Space Society Congratulates Blue Origin and Jeff Bezos for the Spectacular First Crewed Flight of the New Shepard: Well-Tested Suborbital Tourist Rocket Soars to 63 Miles; Opens New Frontiers July 21st, 2021

Scientists create rechargeable swimming microrobots using oil and water July 16th, 2021

Scientists take first snapshots of ultrafast switching in a quantum electronic device: They discover a short-lived state that could lead to faster and more energy-efficient computing devices July 16th, 2021

Researchers discover a new inorganic material with lowest thermal conductivity ever reported July 16th, 2021

Academic/Education

NSF renews Rice-based NEWT Center for water treatment: Partnership primed to introduce game-changing technologies to address global needs October 15th, 2020

Matching Investment Program (MIP) Leverages $140K Empire State Development/NYSTAR Funding to SUNY Polyís CATN2 to Enable $1.5M in Matching Commitments from Industry Partners: Investment Funds Faculty Research Related to Advanced Materials, Genomics, and Semiconductor Reliability October 18th, 2019

A Quantum Leap: $25M grant makes UC Santa Barbara home to the nationís first NSF-funded Quantum Foundry, a center for development of materials for quantum information-based technologies September 16th, 2019

LPU signs MoU with Bruker India for Research Cooperation in Nanotechnology and Material Science September 3rd, 2019

Nanomedicine

The virus trap: Hollow nano-objects made of DNA could trap viruses and render them harmless July 16th, 2021

NYUAD study maps nanobody structure, leading to new ways to potentially fight diseases July 4th, 2021

Optical tweezer technology tweaked to overcome dangers of heat June 25th, 2021

Arrowhead Presents Preclinical Data on ARO-DUX4 at FSHD Society International Research Congress June 25th, 2021

Discoveries

Repairs using light signals: FAU research group develops smart microparticle that identifies defective parts in electrical appliances July 16th, 2021

Removing the lead hazard from perovskite solar cells July 16th, 2021

Scientists create rechargeable swimming microrobots using oil and water July 16th, 2021

Scientists take first snapshots of ultrafast switching in a quantum electronic device: They discover a short-lived state that could lead to faster and more energy-efficient computing devices July 16th, 2021

Announcements

The National Space Society Congratulates Blue Origin and Jeff Bezos for the Spectacular First Crewed Flight of the New Shepard: Well-Tested Suborbital Tourist Rocket Soars to 63 Miles; Opens New Frontiers July 21st, 2021

Scientists create rechargeable swimming microrobots using oil and water July 16th, 2021

Scientists take first snapshots of ultrafast switching in a quantum electronic device: They discover a short-lived state that could lead to faster and more energy-efficient computing devices July 16th, 2021

Researchers discover a new inorganic material with lowest thermal conductivity ever reported July 16th, 2021

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

The National Space Society Congratulates Blue Origin and Jeff Bezos for the Spectacular First Crewed Flight of the New Shepard: Well-Tested Suborbital Tourist Rocket Soars to 63 Miles; Opens New Frontiers July 21st, 2021

The virus trap: Hollow nano-objects made of DNA could trap viruses and render them harmless July 16th, 2021

Removing the lead hazard from perovskite solar cells July 16th, 2021

Scientists create rechargeable swimming microrobots using oil and water July 16th, 2021

Nanobiotechnology

The virus trap: Hollow nano-objects made of DNA could trap viruses and render them harmless July 16th, 2021

Scientists create rechargeable swimming microrobots using oil and water July 16th, 2021

NYUAD study maps nanobody structure, leading to new ways to potentially fight diseases July 4th, 2021

Putting functional proteins in their place: Using DNA-based assembly, scientists developed a method for creating designed and biologically active 2-D and 3-D protein arrays, which show promise for applications in structural biology, biomaterials, nanomedicine, and biocatalysis June 25th, 2021

Research partnerships

Scientists create rechargeable swimming microrobots using oil and water July 16th, 2021

Stress-free path to stress-free metallic films paves the way for next-gen circuitry: Optimized sputtering technique helps minimize stress in tungsten thin films July 4th, 2021

New family of atomic-thin electride materials discovered June 11th, 2021

Magnetism drives metals to insulators in new experiment: Study provides new tools to probe novel spintronic devices June 4th, 2021

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




  Premium Products
NanoNews-Custom
Only the news you want to read!
 Learn More
NanoStrategies
Full-service, expert consulting
 Learn More











ASP
Nanotechnology Now Featured Books




NNN

The Hunger Project