Multi-dose gene therapy for cystic fibrosis; old age proteins in mice and curing hereditary deafness. This week in Abstract Science.

Taking Flight

(Eureka Blog, 7/8/2015, Mary McElroy)

On Thursday July 3, 2015, the UK Cystic Fibrosis Gene Therapy Consortium published the results of their much awaited multi-dose gene therapy clinical trial (PDF) in cystic fibrosis patients—the first trial to evaluate the effectiveness of a genetic cure for this life debilitating disease—and the findings look very positive. Mary McElroy, a Principal Scientist for Charles River in Edinburgh, discussed how in the current multi-dose trial, patients were administered the functioning Cystic Fibrosis Transmembrane Conductance Regulator Protein (CFTR) gene in a non-viral vector (a plasmid) in combination with a liposome vector (known as GL67A). Data from the trial demonstrated that patients administered the gene therapy had a significant, albeit modest, improvement in lung function.

Mice with Hereditary Deafness Hear Again Thanks to Gene Therapy

(New Scientist, 7/8/2015, Andy Coghlan)

At least half of all cases of childhood deafness are caused by inherited conditions. New Scientist reports that a team of scientists, led by Jeffrey Holt of Harvard Medical School, have treated hereditarily deaf mice with a targeted gene therapy, and successfully restored their hearing. Holt’s team focused on TMC1, one of nearly 70 genes which causes hereditary deafness and accounts for 4 to 8 percent of cases. The team equipped a virus, adeno-associated virus-1, with corrected copies of the faulty TMC1 gene, and then injected a solution into the inner ears of mice with the TMC1 mutation. Tests on inner ear cells from treated mice showed that the inserted genes were doing their job. The team is now working on increasing the efficiency of their procedure, determining the length of time the cure will last and are looking at potential applications for hereditary blindness.

‘Old-Age Protein’ May Cause Memory Loss

(Science Magazine, 7/6/2015, Sarah C.P. Williams)

The science behind forgetfulness is becoming clearer, as scientists hone in on a protein in blood that may cause cognitive disorders as it ages. Science Magazine reported on the findings of a study which showed that adding the blood of older mice to younger animal’s bodies makes them sluggish, weaker and more forgetful. Similarly, when the blood of younger models was added to older mice, the restoration of memory and energy can occur. Neuroscientist Saul Villeda of University of California, San Francisco (UCSF) found that the β2 microglobulin (B2M), an immune protein normally involved in distinguishing one’s own cells from invading pathogens may be the cause.

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