Nanotechnology Now

Our NanoNews Digest Sponsors

Heifer International

Wikipedia Affiliate Button

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

Nanometrics Announces Retirement Plans of CEO Timothy Stultz: Dr. Stultz to Continue as Director May 25th, 2017

Nanomechanics, Inc. to Exhibit at the SEM Conference: Nanoindentation experts will attend and exhibit their instruments at the Conference and Exposition on Experimental and Applied Mechanics in Indianapolis May 25th, 2017

Three-dimensional graphene: Experiment at BESSY II shows that optical properties are tuneable May 24th, 2017

Leti to Demo 1st Wireless UNB Transceiver for ‘Massive Internet of Things’ at RFIC 2017 and IMS 2017: Leti Will also Present Three Papers & Two Workshops on 5G Communications IC Design, from RF to mm-Wave, During IMS 2017 and RFIC 2017 in Hawaii May 24th, 2017

Possible Futures

Nanomechanics, Inc. to Exhibit at the SEM Conference: Nanoindentation experts will attend and exhibit their instruments at the Conference and Exposition on Experimental and Applied Mechanics in Indianapolis May 25th, 2017

Three-dimensional graphene: Experiment at BESSY II shows that optical properties are tuneable May 24th, 2017

GLOBALFOUNDRIES and Chengdu Partner to Expand FD-SOI Ecosystem in China: More than $100M investment to establish a center of excellence for FDXTM FD-SOI design May 23rd, 2017

Zap! Graphene is bad news for bacteria: Rice, Ben-Gurion universities show laser-induced graphene kills bacteria, resists biofouling May 22nd, 2017

Academic/Education

MIT Energy Initiative awards 10 seed fund grants for early-stage energy research May 4th, 2017

Bar-Ilan University to set up quantum research center May 1st, 2017

California Research Alliance by BASF establishes more than 25 research projects in three years April 26th, 2017

SUNY Polytechnic Institute Announces Total of 172 Teams Selected to Compete in Solar in Your Community Challenge: Teams from 40 states, plus Washington, DC, 2 Territories, and 4 American Indian Reservations, Will Deploy Solar in Underserved Communities April 20th, 2017

Nanomedicine

Zap! Graphene is bad news for bacteria: Rice, Ben-Gurion universities show laser-induced graphene kills bacteria, resists biofouling May 22nd, 2017

Sensors detect disease markers in breath May 19th, 2017

Oddball enzyme provides easy path to synthetic biomaterials May 17th, 2017

The brighter side of twisted polymers: Conjugated polymers designed with a twist produce tiny, brightly fluorescent particles with broad applications May 16th, 2017

Discoveries

Three-dimensional graphene: Experiment at BESSY II shows that optical properties are tuneable May 24th, 2017

Zap! Graphene is bad news for bacteria: Rice, Ben-Gurion universities show laser-induced graphene kills bacteria, resists biofouling May 22nd, 2017

Sensors detect disease markers in breath May 19th, 2017

Graphene-nanotube hybrid boosts lithium metal batteries: Rice University prototypes store 3 times the energy of lithium-ion batteries May 19th, 2017

Announcements

Nanometrics Announces Retirement Plans of CEO Timothy Stultz: Dr. Stultz to Continue as Director May 25th, 2017

Nanomechanics, Inc. to Exhibit at the SEM Conference: Nanoindentation experts will attend and exhibit their instruments at the Conference and Exposition on Experimental and Applied Mechanics in Indianapolis May 25th, 2017

Three-dimensional graphene: Experiment at BESSY II shows that optical properties are tuneable May 24th, 2017

Leti to Demo 1st Wireless UNB Transceiver for ‘Massive Internet of Things’ at RFIC 2017 and IMS 2017: Leti Will also Present Three Papers & Two Workshops on 5G Communications IC Design, from RF to mm-Wave, During IMS 2017 and RFIC 2017 in Hawaii May 24th, 2017

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

Zap! Graphene is bad news for bacteria: Rice, Ben-Gurion universities show laser-induced graphene kills bacteria, resists biofouling May 22nd, 2017

Gas gives laser-induced graphene super properties: Rice University study shows inexpensive material can be superhydrophilic or superhydrophobic May 15th, 2017

Fed grant backs nanofiber development: Rice University joins Department of Energy 'Next Generation Machines' initiative May 10th, 2017

'Hot' electrons don't mind the gap: Rice University scientists find nanogaps in plasmonic gold wires enhance voltage when excited May 8th, 2017

Nanobiotechnology

Zap! Graphene is bad news for bacteria: Rice, Ben-Gurion universities show laser-induced graphene kills bacteria, resists biofouling May 22nd, 2017

Sensors detect disease markers in breath May 19th, 2017

Oddball enzyme provides easy path to synthetic biomaterials May 17th, 2017

The brighter side of twisted polymers: Conjugated polymers designed with a twist produce tiny, brightly fluorescent particles with broad applications May 16th, 2017

Research partnerships

Three-dimensional graphene: Experiment at BESSY II shows that optical properties are tuneable May 24th, 2017

Zap! Graphene is bad news for bacteria: Rice, Ben-Gurion universities show laser-induced graphene kills bacteria, resists biofouling May 22nd, 2017

Sensors detect disease markers in breath May 19th, 2017

Stanford scientists use nanotechnology to boost the performance of key industrial catalyst May 18th, 2017

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