Nanotechnology Now

Our NanoNews Digest Sponsors





Heifer International

Wikipedia Affiliate Button


android tablet pc

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

Sunblock poses potential hazard to sea life August 20th, 2014

Rice physicist emerges as leader in quantum materials research: Nevidomskyy wins both NSF CAREER Award and Cottrell Scholar Award August 20th, 2014

Graphene may be key to leap in supercapacitor performance August 20th, 2014

Newly-Developed Nanobiosensor Quickly Diagnoses Cancer August 20th, 2014

Possible Futures

Air Force’s 30-year plan seeks 'strategic agility' August 1st, 2014

IBM Announces $3 Billion Research Initiative to Tackle Chip Grand Challenges for Cloud and Big Data Systems: Scientists and engineers to push limits of silicon technology to 7 nanometers and below and create post-silicon future July 10th, 2014

Virus structure inspires novel understanding of onion-like carbon nanoparticles April 10th, 2014

Local girl does good March 22nd, 2014

Academic/Education

SEMATECH and Newly Merged SUNY CNSE/SUNYIT Launch New Patterning Center to Further Advance Materials Development: Center to Provide Access to Critical Tools that Support Semiconductor Technology Node Development August 7th, 2014

Oxford Instruments Asylum Research and the Center for Nanoscale Systems at Harvard University Present a Workshop on AFM Nanomechanical and Nanoelectrical Characterization, Aug. 21-22 August 6th, 2014

University of Manchester selects Anasys AFM-IR for coatings and corrosion research July 30th, 2014

Haydale Announces Collaboration Agreement with Swansea University’s Welsh Centre for Printing and Coatings (WCPC) July 12th, 2014

Nanomedicine

Newly-Developed Nanobiosensor Quickly Diagnoses Cancer August 20th, 2014

Graphene rubber bands could stretch limits of current healthcare, new research finds August 19th, 2014

Interaction between Drug, DNA for Designing Anticancer Drugs Studied in Iran August 17th, 2014

Iranian Scientists Stabilize Protein on Highly Stable Electrode Surface August 14th, 2014

Discoveries

Sunblock poses potential hazard to sea life August 20th, 2014

Rice physicist emerges as leader in quantum materials research: Nevidomskyy wins both NSF CAREER Award and Cottrell Scholar Award August 20th, 2014

Newly-Developed Nanobiosensor Quickly Diagnoses Cancer August 20th, 2014

Ultrasonic Waves Applied in Production of Graphene Nanosheets August 20th, 2014

Announcements

Rice physicist emerges as leader in quantum materials research: Nevidomskyy wins both NSF CAREER Award and Cottrell Scholar Award August 20th, 2014

Graphene may be key to leap in supercapacitor performance August 20th, 2014

Newly-Developed Nanobiosensor Quickly Diagnoses Cancer August 20th, 2014

Ultrasonic Waves Applied in Production of Graphene Nanosheets August 20th, 2014

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

Rice physicist emerges as leader in quantum materials research: Nevidomskyy wins both NSF CAREER Award and Cottrell Scholar Award August 20th, 2014

Oxford Instruments Asylum Research Receives the 2014 Microscopy Today Innovation Award for blueDrive Photothermal Excitation August 18th, 2014

AQUANOVA receives Technology Leadership Award 2014 FROST & SULLIVAN honors NovaSOL® Technology again August 12th, 2014

Focal blood-brain-barrier disruption with high-frequency pulsed electric fields August 12th, 2014

Nanobiotechnology

The channel that relaxes DNA: Relaxing DNA strands by using nano-channels: Instructions for use August 20th, 2014

Сalculations with Nanoscale Smart Particles August 19th, 2014

Interaction between Drug, DNA for Designing Anticancer Drugs Studied in Iran August 17th, 2014

Scientists fold RNA origami from a single strand: RNA origami is a new method for organizing molecules on the nanoscale. Using just a single strand of RNA, this technique can produce many complicated shapes. August 14th, 2014

Research partnerships

Сalculations with Nanoscale Smart Particles August 19th, 2014

Promising Ferroelectric Materials Suffer From Unexpected Electric Polarizations: Brookhaven Lab scientists find surprising locked charge polarizations that impede performance in next-gen materials that could otherwise revolutionize data-driven devices August 18th, 2014

Nano Bonds Increase Raw Strength of Fireproof Concretes August 18th, 2014

Production of Toxic Ion Nanosorbents with High Sorption Capacity in Iran August 17th, 2014

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







© Copyright 1999-2014 7th Wave, Inc. All Rights Reserved PRIVACY POLICY :: CONTACT US :: STATS :: SITE MAP :: ADVERTISE