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

Reversible Writing with Light: Self-assembling nanoparticles take their cues from their surroundings September 3rd, 2015

For 2-D boron, it's all about that base: Rice University theorists show flat boron form would depend on metal substrates September 2nd, 2015

Silk bio-ink could help advance tissue engineering with 3-D printers September 2nd, 2015

Phagraphene, a 'relative' of graphene, discovered September 2nd, 2015

Possible Futures

Silk bio-ink could help advance tissue engineering with 3-D printers September 2nd, 2015

Sediment dwelling creatures at risk from nanoparticles in common household products August 13th, 2015

Harris & Harris Group Reports Financial Statements as of June 30, 2015, and Announces a Stock Repurchase Program August 10th, 2015

Molecular trick alters rules of attraction for non-magnetic metals August 5th, 2015

Academic/Education

Sustainable nanotechnology center September 1st, 2015

National Science Foundation Selects SUNY Poly CNSE for Expanded $2.1M Northeast Advanced Technological Education Center: NSF Center Locates to NanoCollege in Support of Flourishing Tech Industry in NYS September 1st, 2015

Announcing Oxford Instruments and School of Physics signing a Memorandum of Understanding August 26th, 2015

Kwansei Gakuin University in Hyogo, Japan, uses Raman microscopy to study crystallographic defects in silicon carbide wafers August 25th, 2015

Nanomedicine

Reversible Writing with Light: Self-assembling nanoparticles take their cues from their surroundings September 3rd, 2015

Silk bio-ink could help advance tissue engineering with 3-D printers September 2nd, 2015

Using DNA origami to build nanodevices of the future September 1st, 2015

Efficiency of Nanodrug Containing Antibiotics in Treatment of Infectious Diseases Evaluated August 31st, 2015

Discoveries

Reversible Writing with Light: Self-assembling nanoparticles take their cues from their surroundings September 3rd, 2015

For 2-D boron, it's all about that base: Rice University theorists show flat boron form would depend on metal substrates September 2nd, 2015

Silk bio-ink could help advance tissue engineering with 3-D printers September 2nd, 2015

Phagraphene, a 'relative' of graphene, discovered September 2nd, 2015

Announcements

Reversible Writing with Light: Self-assembling nanoparticles take their cues from their surroundings September 3rd, 2015

Silk bio-ink could help advance tissue engineering with 3-D printers September 2nd, 2015

Phagraphene, a 'relative' of graphene, discovered September 2nd, 2015

A marine creature's magic trick explained: Crystal structures on the sea sapphire's back appear differently depending on the angle of reflection September 2nd, 2015

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

Reversible Writing with Light: Self-assembling nanoparticles take their cues from their surroundings September 3rd, 2015

A marine creature's magic trick explained: Crystal structures on the sea sapphire's back appear differently depending on the angle of reflection September 2nd, 2015

RUSNANOPRIZE Directorate Announces New Deadline for Nominations Submission September 11, 2015 September 1st, 2015

$200K Awarded to Develop In Vitro Lung Test for Toxicity of Inhaled Nanomaterials: In Vitro Lung Test Designed to Protect Human Health and Replace Animal Testing September 1st, 2015

Nanobiotechnology

A marine creature's magic trick explained: Crystal structures on the sea sapphire's back appear differently depending on the angle of reflection September 2nd, 2015

Using DNA origami to build nanodevices of the future September 1st, 2015

Researchers use DNA 'clews' to shuttle CRISPR-Cas9 gene-editing tool into cells August 30th, 2015

Small but heading for the big time: Nanobiotix half year results for the six months ended 30 June 2015, in line with expectations: Major clinical achievements and corporate developments August 28th, 2015

Research partnerships

Turning clothing into information displays September 2nd, 2015

Sustainable nanotechnology center September 1st, 2015

$200K Awarded to Develop In Vitro Lung Test for Toxicity of Inhaled Nanomaterials: In Vitro Lung Test Designed to Protect Human Health and Replace Animal Testing September 1st, 2015

Hot electrons point the way to perfect light absorption: Physicists study how to achieve perfect absorption of light with the help of rough ultrathin films September 1st, 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







Car Brands
Buy website traffic