gene therapy

Discovery shows fat triggers rheumatoid arthritis
05/08/2013 - 10:06

Scientists have discovered that fat cells in the knee secrete a protein linked to arthritis, a finding that paves the way for new gene therapies that could offer relief and mobility to millions worldwide.

 

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Gene Therapy for Human Disease: Clinical Advances and Challenges
03/23/2013 - 11:03

The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today. Research articles included in the special issue highlight current studies that, after decades of trial and error, may provide evidence for a clear path of treatment and cure for many diseases. There are more than 1,800 genetic disorders known in humans, and only a small fraction of these can be treated and even fewer cured. Some of these disorders are exceedingly rare, others more common. The approach of gene therapy however may be applicable to all.

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Exon Skipping to Restore Gene Expression Is Promising Therapeutic Strategy for Muscular Dystrophy
01/15/2013 - 15:11

A novel therapeutic approach called exon skipping involves bypassing a disease-causing mutation in a gene to restore normal gene expression and protein production. Two innovative examples of this strategy used to correct gene defects associated with muscular dystrophy are described in articles in Human Gene Therapy.

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Researchers test new gene therapy method in human cells...and it works
10/25/2012 - 09:34

Oregon Health & Science University's development of a new gene therapy method to prevent certain inherited diseases has reached a significant milestone. Researchers at the university's Oregon National Primate Research Center and the OHSU Department of Obstetrics & Gynecology have successfully demonstrated their procedure in human cells. It's believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects.

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Researchers Halt Autoimmune Disease Myasthenia Gravis in Mice
10/01/2012 - 10:30

Working with mice, Johns Hopkins researchers say they have developed a gene-based therapy to stop the rodent equivalent of the autoimmune disease myasthenia gravis by specifically targeting the destructive immune response the disorder triggers in the body.

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Immune systems of 'bubble babies' restored by gene therapy, UCLA researchers find
09/11/2012 - 14:02

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "bubble boy" disease, a life-threatening condition that if left untreated can be fatal within one to two years.

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