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New Gene Delivery Tool May Help Develop Therapies For Macular Degeneration

January 28, 2010

Adapted from Tufts University

Researchers at Tufts University School of Medicine (TUSM) and the Sackler School of Graduate Biomedical Sciences at Tufts have developed a new tool for gene therapy that significantly increases gene delivery to cells in the retina compared to other carriers and DNA alone, according to a study published in the January issue of The Journal of Gene Medicine. The tool, a peptide called PEG-POD, provides a vehicle for therapeutic genes and may help researchers develop therapies for degenerative eye disorders such as retinitis pigmentosa and age-related macular degeneration.

Safe and effective delivery of therapeutic genes has been a major obstacle in gene therapy research. Deactivated viruses have frequently been used in the past, but concerns about the safety of this method have left scientists seeking new ways to get therapeutic genes into cells. PEG-POD, the new gene delivery tool, may be the answer.

“We think the level of gene expression seen with PEG-POD may be enough to protect the retina from degeneration, slowing the progression of eye disorders and we have preliminary evidence that this is indeed the case,” said co-author Siobhan Cashman, Ph.D., research assistant professor in the department of ophthalmology at TUSM and member of lab run by senior authro, Rajendra Kumar-Singh, Ph.D., associate professor of ophthalmology and adjunct associate professor of neuroscience at Tufts University School of Medicine.

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