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

Fast, efficient sperm tails inspire nanobiotechnology December 5th, 2016

Journal Nanotechnology Progress International (JONPI) Volume 6, issue 2 coming out soon! December 5th, 2016

Infrared instrumentation leader secures exclusive use of Vantablack coating December 5th, 2016

Construction of practical quantum computers radically simplified: Scientists invent ground-breaking new method that puts quantum computers within reach December 5th, 2016

Possible Futures

Fast, efficient sperm tails inspire nanobiotechnology December 5th, 2016

Construction of practical quantum computers radically simplified: Scientists invent ground-breaking new method that puts quantum computers within reach December 5th, 2016

Shape matters when light meets atom: Mapping the interaction of a single atom with a single photon may inform design of quantum devices December 4th, 2016

UTSA study describes new minimally invasive device to treat cancer and other illnesses: Medicine diffusion capsule could locally treat multiple ailments and diseases over several weeks December 3rd, 2016

Academic/Education

Oxford Nanoimaging report on how the Nanoimager, a desktop microscope delivering single molecule, super-resolution performance, is being applied at the MRC Centre for Molecular Bacteriology & Infection November 22nd, 2016

The University of Applied Sciences in Upper Austria uses Deben tensile stages as an integral part of their computed tomography research and testing facility October 18th, 2016

Enterprise In Space Partners with Sketchfab and 3D Hubs for NewSpace Education October 13th, 2016

New Agricultural Research Center Debuts at UCF October 12th, 2016

Nanomedicine

Fast, efficient sperm tails inspire nanobiotechnology December 5th, 2016

Journal Nanotechnology Progress International (JONPI) Volume 6, issue 2 coming out soon! December 5th, 2016

UTSA study describes new minimally invasive device to treat cancer and other illnesses: Medicine diffusion capsule could locally treat multiple ailments and diseases over several weeks December 3rd, 2016

Nanobiotix Provides Update on Global Development of Lead Product NBTXR3: Seven clinical trials across the world: More than 2/3 of STS patients recruited in the “act.in.sarc” Phase II/III trial: Phase I/II prostate cancer trial now recruiting in the U.S. November 28th, 2016

Discoveries

Fast, efficient sperm tails inspire nanobiotechnology December 5th, 2016

Construction of practical quantum computers radically simplified: Scientists invent ground-breaking new method that puts quantum computers within reach December 5th, 2016

Shape matters when light meets atom: Mapping the interaction of a single atom with a single photon may inform design of quantum devices December 4th, 2016

UTSA study describes new minimally invasive device to treat cancer and other illnesses: Medicine diffusion capsule could locally treat multiple ailments and diseases over several weeks December 3rd, 2016

Announcements

Fast, efficient sperm tails inspire nanobiotechnology December 5th, 2016

Journal Nanotechnology Progress International (JONPI) Volume 6, issue 2 coming out soon! December 5th, 2016

Infrared instrumentation leader secures exclusive use of Vantablack coating December 5th, 2016

Construction of practical quantum computers radically simplified: Scientists invent ground-breaking new method that puts quantum computers within reach December 5th, 2016

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

Shape matters when light meets atom: Mapping the interaction of a single atom with a single photon may inform design of quantum devices December 4th, 2016

Quantum obstacle course changes material from superconductor to insulator December 1st, 2016

'Back to the Future' inspires solar nanotech-powered clothing November 15th, 2016

2-D material a brittle surprise: Rice University researchers finds molybdenum diselenide not as strong as they thought November 14th, 2016

Nanobiotechnology

Fast, efficient sperm tails inspire nanobiotechnology December 5th, 2016

Deep insights from surface reactions: Researchers use Stampede supercomputer to study new chemical sensing methods, desalination and bacterial energy production December 2nd, 2016

Nanobiotix Provides Update on Global Development of Lead Product NBTXR3: Seven clinical trials across the world: More than 2/3 of STS patients recruited in the “act.in.sarc” Phase II/III trial: Phase I/II prostate cancer trial now recruiting in the U.S. November 28th, 2016

From champagne bubbles, dance parties and disease to new nanomaterials: Understanding nucleation of protein filaments might help with Alzheimer's Disease and type 2 Diabetes November 24th, 2016

Research partnerships

Deep insights from surface reactions: Researchers use Stampede supercomputer to study new chemical sensing methods, desalination and bacterial energy production December 2nd, 2016

Quantum obstacle course changes material from superconductor to insulator December 1st, 2016

Novel silicon etching technique crafts 3-D gradient refractive index micro-optics November 28th, 2016

Single photon converter -- a key component of quantum internet November 28th, 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