Category Archives: Gene Therapy

Fred Dufour/AFP/Getty Images Electronic pacemakers require invasive surgery to install and carry the risk of infection. Gene therapy could soon join the electronic pacemaker as a treatment for a weak heart. By inserting a specific gene into heart-muscle cells, researchers can restore a normal heart rate in pigs at least temporarily. Electronic pacemakers restore regular function to slowing and arrhythmic hearts by using electricity to stimulate their beating a function usually peformed by the sinoatrial node, a cluster of a few thousand cardiac cells that signal the heart to pump at a regular rate. Although implanted pacemakers are widely used, they require an invasive surgery to be installed, carry the risk of infection, and can set off alarms during airport security checks. To overcome these limitations, a team led by Eduardo Marbn, a cardiologist at Cedars-Sinai Medical Center in Los Angeles, California, sought to coax heart cells outside the sinoatrial node to keep the beat using a less invasive approach. The findings are reported today in Science Translational Medicine1. In 12 pigs, the team mimicked a fatal human heart condition in which electrical activity cannot spread through the heart from the sinoatrial node, forcing other, weaker parts of the heart … Continue reading →

NEW YORK, July 16 (UPI) --Pacemakers are one of modern medicine's great inventions. The mechanical timekeeping device essentially salvages failing hearts, saving 3,000 lives every year. Despite their accomplishments, scientists have been looking for an alternative: A way to avoid the invasive surgery -- and the associated risks -- required by traditional pacemakers. A new gene therapy technique suggests humans may be able to just grow their own. While the technique hasn't yet been tested on humans, it has offered promising results in experiments on mice and now, most recently, on pigs. The technique is as follows: scientists inject a gene called TBX18 into heart muscle cells. The gene causes normal heart cells to transform into the type of special cells that dictate the speed of the pumping heart. The genes actually aren't injected directly into the heart, but are loaded onto deactivated cold viruses which infect the heart cells and deliver the new code. "This development heralds a new era in gene therapy, where genes are used not only to correct deficiency disorders but to convert one cell to another to treat disease," said Dr. Eduardo Marban -- director of the Cedars-Sinai Heart Institute, in New York, and author … Continue reading →

Researchers say they've found a way to transform ordinary pig heart muscle cells into a "biological pacemaker," a feat that might one day lead to the replacement of electronic pacemakers in humans. "Rather than having to undergo implantation with a metallic device that needs to be replaced regularly and can fail or become infected, patients may someday be able to undergo a single gene injection and be cured of slow heart rhythm forever," said senior study author Dr. Eugenio Cingolani, director of the Cedars-Sinai Heart Institute's Cardiogenetics-Familial Arrhythmia Clinic, in Los Angeles. Using gene therapy, the researchers altered a peppercorn-sized area in the heart muscle of pigs to create a new "sino-atrial node" -- the bundle of neurons that normally serves as the heart's natural pacemaker. The technique kept alive a handful of pigs suffering from complete heart block, a condition in which the heart beats very slowly or not at all due to problems in the heart's electrical system. The biological pacemaker also appeared to function as well as an original sino-atrial node and better than typical electronic pacemakers, said study co-author Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute, in Los Angeles. "When we exercise, our hearts … Continue reading →

Washington No batteries required: Scientists are creating a biological pacemaker by injecting a gene into the hearts of sick pigs that changed ordinary cardiac cells into a special kind that induces a steady heartbeat. The study, published Wednesday, is one step toward developing an alternative to electronic pacemakers that are implanted into 300,000 Americans a year. There are people who desperately need a pacemaker but cant get one safely, said Dr. Eduardo Marban, director of the Cedars-Sinai Heart Institute in Los Angeles, who led the work. This development heralds a new era of gene therapy that one day might offer them an option. Your heartbeat depends on a natural pacemaker, a small cluster of cells its about the size of a peppercorn, Marban says that generates electrical activity. Called the sinoatrial node, it acts like a metronome to keep the heart pulsing at 60 to 100 beats a minute or so, more when youre active. If that node quits working correctly, hooking the heart to an electronic pacemaker works very well for most people. But about 2 percent of recipients develop an infection that requires the pacemaker to be removed for weeks until antibiotics wipe out the germs, Marban said. … Continue reading →

WASHINGTON (Reuters) - Researchers have succeeded in turning ordinary cardiac muscle cells into specialized ones that deliver a steady heartbeat using a gene therapy procedure they predict could become an alternative to implanted electronic pacemakers. A study published on Wednesday involved pigs with a condition called heart block that makes their hearts beat too slowly. By injecting a human gene into a tiny region of the heart's pumping chambers roughly the size of a peppercorn, the researchers reprogrammed heart muscle cells into a type of cell that emits electrical impulses to drive the beating heart. In doing so, cardiologists at Cedars-Sinai Heart Institute in Los Angeles created "biological pacemaker" cells that restored a normal heart rate in the pigs. The procedure achieved the same result as implanting an electronic pacemaker that sends electrical pulses to the heart if it beats too slowly or skips a beat. "This development heralds a new era of gene therapy where genes are used not only to correct a deficiency disorder but actually to convert one type of cell into another to treat disease," Dr. Eduardo Marbn, director of the Cedars-Sinai Heart Institute and leader of the research team, told reporters. The researchers noted that … Continue reading →

Syal, Tiffany, Wintergalen, Wu, Zautke Group Pre Life Project on Gene Therapy This is our group project on Gene Therapy for Summer Bioethics. By: Connor Zautke … Continue reading →

Advances in plan for individual gene therapies for leading cause of blindness Scientists use stem cells to investigate causes of degenerative eye disease Two patients with retinitis pigmentosa had mutations in a certain gene New gene therapy was used to rescue vision of mice with the same condition By Julian Robinson Published: 06:49 EST, 11 July 2014 | Updated: 08:08 EST, 11 July 2014 212 shares 3 View comments Scientists have moved closer to a breakthrough in 'personalised' treatments for a leading cause of blindness. Researchers have stepped up their bid to create individual gene therapies for one of the factors that triggers inherited vision loss. They used 'induced' stem cells - taken from ordinary skin cells - to investigate patient-specific causes of the degenerative eye disease retinitis pigmentosa (RP), which leads to blindness or severe visual impairment. Scientists have moved closer to a breakthrough in 'personalised' treatments for a leading cause of blindness Link: Breakthrough in bid to cure blindness as scientists identify gene … Continue reading →

Cell and Gene Therapy Center By: RowanSOM … Continue reading →

The general approach of gene therapy is nothing more than an extension of the technique for clone selection by functional complementation (Chapter 12). The functions absent in the recipient as a result of a defective gene are introduced on a vector that inserts into one of the recipients chromosomes and thereby generates a transgenic animal that has been genetically cured. The technique is of great potential in humans because it offers the hope of correcting hereditary diseases. However, gene therapy is also being applied to mammals other than humans. The first example of gene therapy in a mammal was the correction of a growth-hormone deficiency in mice. The recessive mutation little (lit) results in dwarf mice. Even though a mouses growth-hormone gene is present and apparently normal, no mRNA for this gene is produced. The initial step in correcting this deficiency was to inject homozygous lit/lit eggs with about 5000 copies of a 5-kb linear DNA fragment that contained the rat growth-hormone structural gene (RGH) fused to a regulatorpromoter sequence from a mouse metallothionein gene (MP). The normal job of metallothionein is to detoxify heavy metals, so the regulatory sequence is responsive to the presence of heavy metals in the … Continue reading →

PUBLIC RELEASE DATE: 7-Jul-2014 Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research Because the adult mammalian central nervous system has only limited intrinsic capacity to regenerate connections after injury, due to factors both intrinsic and extrinsic to the mature neuron, therapies are required to support the survival of injured neurons and to promote the long-distance regrowth of axons back to their original target structures. The retina and optic nerve are part of the CNS and this system is much used in experiments designed to test new ways of promoting regeneration after injury. Testing of therapies designed to improve RGCs viability also has direct clinical relevance because there is loss of these centrally projecting neurons in many ophthalmic diseases. Many different approaches are being trialed, targeting different receptor systems and/or different signaling pathways, some aimed at enhancing intrinsic growth capacity in injured RGCs, others aimed at reducing the impact of factors external to the neuron that suppress/restrict the regenerative response. An approach increasingly of interest involves the use of modified, replication-deficient viral vectors to introduce appropriate genes into injured cells in the visual pathway (gene therapy). In the perspective article written by Prof Alan Harvey, from School of Anatomy, Physiology and … Continue reading →