Researchers restore the sight of blind mice using gene therapy

University of Florida researchers used gene therapy to restore sight in mice with a form of hereditary blindness, a finding that has bearing on many of the most common blinding diseases.

Writing online in today’s (May 21) edition of Nature Medicine, scientists describe how they used a harmless virus to deliver corrective genes to mice with a genetic impairment that robs them of vision.

The discovery shows that it is possible to target and rescue cone cells — the most important cells for visual sharpness and color vision in people.

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Antibiotic inhibits cancer gene activity

A little-known antibiotic shows early promise as an anti-cancer agent, inhibiting a gene found at higher-than-normal levels in most human tumors, according to researchers at the University of Illinois at Chicago College of Medicine.

Jumping gene could provide non-viral alternative for gene therapy

A jumping gene first identified in a cabbage-eating moth may one day provide a safer, target-specific alternative to viruses for gene therapy, researchers say.

They compared the ability of the four best-characterized jumping genes, or transposons, to insert themselves into a cell’s DNA and produce a desired change, such as making the cell resistant to damage from radiation therapy.

They found the piggyBac transposon was five to 10 times better than the other circular pieces of DNA at making a home and a difference in several mammalian cell lines, including three human ones.

Reprogramming Biology

Visionary futurist Ray Kurzweil, whose remarkable ideas on technological progress have been an inspiration for Biosingularity blogs, have a wonderful concise article on biological advances in recent issue of Scientific American. 

As a scientist working on biological systems I fully agree and whole heartedly support Kurzweil's observations that: " Biology is now in the early stages of an historic transition to an information science, while also gaining the tools to reprogram the ancient information systems of life ….. We are now beginning to understand biology as a set of information processes, and we're developing realistic models and simulations of how the processes involved in disease and aging progress. Moreover, we are developing the tools to reprogram them."

In the article Kurzweil predicts that tinkering with our genetic programs will extend human lifespan beyond the current limits. He also reiterates that biological systems are also subject to the "law of accelerating returns", which had tremendous impact on information technologies. Indeed, the cost of sequencing and synthesizing gene base pairs have decreased more than 10,000 fold over the last 15 years, and this exponential progress is currently accelerating as predicted by Kurzweil in his recent book. 

Read rest of the article at Scientific American web site.
 

Researchers develop all-in-one remote control gene expression tool derived from HIV

In an article appearing online today in the journal Nature Methods, researchers at the EPFL (Ecole Polytechnique Fédérale de Lausanne) unveil a powerful new tool that will facilitate genetic research and open up new avenues for the clinical treatment of genetic disease.

An all-in-one tool like this – efficiently combining techniques that each previously required separate delivery – will likely see wide use in genetic research and in clinical gene therapy applications. It is particularly applicable for use in stem cells, embryonic cells and tissues and organs that are amenable to genetic transduction.

Sickle cell disease corrected in human models using stem cell-based gene therapy

In a study to be published in the January 2006 issue of Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a novel strategy that uses stem cell-based gene therapy and RNA interference to genetically reverse sickle cell disease (SCD) in human cells. This research is the first to demonstrate a way to genetically correct this debilitating blood disease using RNA interference technology.

Researchers hone in on differentiation of heart stem cells

A team of scientists from the Gladstone Institute of Cardiovascular Disease (GICD) has identified a key factor in heart development that could help advance gene therapy for treating cardiac disorders.

The findings could help cardiac stem cell researchers one day develop strategies for gene and cell- mediated cardiac therapies.

Researchers Demonstrate A New Way To Switch Therapeutic Genes ‘On’ And ‘Off’

A gene therapy research team at Cedars-Sinai Medical Center has developed a new method of signaling therapeutic genes to turn “off” or “on,” a mechanism that could enable scientists to fine-tune genetic- and stem cell-based therapies so that they are safer, more controllable and more effective.

Although other similar signaling systems have been developed, the Cedars-Sinai research is the first to give physicians the flexibility to arbitrarily turn the gene expression on or off even in the presence of an immune response to adenovirus, as would be present in most patients undergoing clinical trials. This has been a major obstacle in bringing the testing of genetic therapies to humans in a clinical setting.