Category Archives: Gene Therapy

A major hurdle in gene therapy is the efficient integration of a corrected gene into a patient's genome without mutating off-target sites. In a paper published today in Genome Research, scientists have used CRISPR/Cas genome editing technology to seamlessly and efficiently correct disease-causing mutations in cells from patients with -thalassemia. -thalassemia results from inherited DNA mutations in the hemoglobin beta (HBB) gene, resulting in reduced HBB expression in red blood cells and, in the most severe forms, anemia. The only established curative treatment is hematopoietic stem cell transplantation; however, this treatment requires a matched donor. Gene therapy, which delivers a corrected copy of a gene into patient cells, could bypass the need for a donor. Previous attempts using a virus to randomly insert a normal gene into the genome has been successful in one -thalassemia patient, but the long-term effect of viral insertion is not yet known. To correct HBB mutations directly in a patient's genome, researchers first generated induced pluripotent stem cells, or iPSCs, from skin cells of patients. The real breakthrough came when they applied CRISPR/Cas9 to precisely engineer a double strand DNA break at the HBB locus in these cells, allowing a donor plasmid with the corrected … Continue reading →

X-men gene therapy session (ASMR) My first attempt at an ASMR video after lurking for so long. A huge thanks to Heatherfeather for making her amazing videos and answering all of my messages. You are the very first test subject... By: Legendoflink … Continue reading →

Treating Disease in Babies Before Birth: The Third Option - Anna David at TEDxUCLWomen Is there a way to treat disease in babies before birth? In this informative talk, Anna David, Associate Professor at the UCL Institute for Women's Health, discusses how foetal gene therapy... By: TEDx Talks … Continue reading →

What is gene therapy? Robin Ali, BSc, PhD, FMedSci, internationally known for his research in gene and cell-based therapy for the treatment of retinal degeneration, has joined the... By: Kellogg Eye Center - Ann Arbor … Continue reading →

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods. In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene. A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common type of vectors are viruses that have been genetically altered to carry normal human DNA. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to harness this ability by manipulating the viral genome to remove disease-causing genes and insert therapeutic ones. Target cells such as the patient's liver or lung cells are infected with the vector. The vector then unloads its genetic material containing the therapeutic human gene into the target cell. Read more from the original … Continue reading →

Pacemakers have long been a life-saving device for people whose hearts dont keep the right beat but they can sometimes come with complications. Now, a team of researchers at the Cedars-Sinai Heart Institute in Los Angeles have managed to transform regular heart cells in pigs into pacemaker cells cells that naturally keep the hearts rhythm in step. The experiments described in the journal Science Translational Medicine mark the first study of this gene therapy in a large animal one on par with human scale. The research could help lead to therapies for people who have to have their electronic pacemakers temporarily removed because of infection and could one day remove the need for surgically implanted pacemaker devices altogether. The heart beats to the sound of its own drum. It relies on a smaller number of specialized pacemaker cells in a tiny region of the heart called the sinoatrial node they are the metronome for the heart. But sometimes the heart can't hear its own timekeeper. It beats too slowly or irregularly and this can lead to dangerous consequences, from fatigue to circulatory collapse. The researchers found a way around this problem: Create new pacemaker cells in a different part of … Continue reading →

By Dennis Thompson HealthDay Reporter WEDNESDAY, July 16, 2014 (HealthDay News) -- 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 … Continue reading →

Health: An Important Development For Heart Patients Who Need Pacemakers Stephanie Stahl has more on what scientists are calling a new era of gene therapy. Official Site: http://philadelphia.cbslocal.com/ Subscribe on YouTube: htt... By: CBS Philly … Continue reading →

A pacemaker that may no longer be needed if the new gene therapy technique works. By Rachael Rettner, Senior Writer for LiveScience 2014-07-17 22:15:37 UTC Electronic pacemakers can be lifesaving for people with abnormal or slow heart rhythms, but not everyone who needs a pacemaker is able to have an electronic device implanted in their heart. Now, in experiments in pigs, researchers have come up with a new method for making a "biological pacemaker" that might one day serve as an alternative to electronic ones, the researchers said. Making this pacemaker involves injecting a gene into heart muscle cells, which transforms these normal heart cells into special cells that can initiate a heartbeat. This method could be useful for certain patients, such as those who develop infections from electronic pacemakers and need to have the devices temporarily removed, or fetuses with life-threatening heart disorders who cannot have an electronic pacemaker implanted, the researchers said. "Babies still in the womb cannot have a pacemaker," study researcher Dr. Eugenio Cingolani, director of the Cardiogenetics-Familial Arrhythmia Clinic at Cedars-Sinai Heart Institute in Los Angeles, said in a statement. "It is possible that one day, we might be able to save lives by replacing … Continue reading →

WASHINGTON -- 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 can't 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 - it's 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 you're 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. And some fetuses are at risk of stillbirth when their heartbeat falters, a condition called congenital heart block. For over a decade, teams of researchers have worked to create a … Continue reading →