Category Archives: Gene Therapy

Science Advances : Gene therapy rescues disease phenotype in a spinal muscular atrophy...... Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model. Monica Nizzardo et al (2015), Scie... By: KeSimpulan … Continue reading →

Gene Therapy Gastrointestinal Insight: Trends and Challenges Analysed in Research Report Gene Therapy Gastrointestinal Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy ... By: James Jacob … Continue reading →

Stamford, CT (PRWEB) March 16, 2015 Alliance for Cancer Gene Therapy (ACGT) the nations only nonprofit dedicated exclusively to cell and gene therapies for cancer has achieved a major milestone, surpassing $25 million donated to innovative and breakthrough cancer research. ACGT was founded by Barbara Netter and her late husband, Edward, in 2001 with the goal of transforming cancer into a manageable, treatable disease. Pushing the foundation across the $25 million threshold are a pair of three-year, $250,000 grants to two esteemed scientists: Meenakshi Hegde, MD, of Texas Childrens Cancer Center at Baylor College of Medicine in Houston, TX, and Christopher Jewell, PhD, at University of Maryland, College Park. Dr. Hegdes work will focus on immunotherapy, specifically adoptive cellular therapy for melanoma. Dr. Jewells research is centered on harnessing intra-lymph node gene therapy to promote tumor immunity. The grantees will develop genetically-modified T cells and cancer vaccines with the potential to stop cancer in its tracks. Drs. Hegde and Jewell are two outstanding scientists in the vanguard of treating and defeating cancer, said Barbara Netter, ACGTs President. Their work offers tremendous hope to those battling cancer, and also to their loved ones. ACGT grants are awarded to promising researchers whose … Continue reading →

Gene Therapy Oncology Insight: Trends and Challenges Analysed in Research Report Gene Therapy Oncology Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy profiles core. By: James Jacob … Continue reading →

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding. Thus far, the new therapy has proven safe. "The result was stunning," said Timothy Nichols, MD, director of the Francis Owen Blood Research Laboratory at the University of North Carolina School of Medicine and co-senior author of the paper. "Just a small amount of new factor IX necessary for proper clotting produced a major reduction in bleeding events. It was extraordinarily powerful." The idea behind gene therapy is that doctors could give hemophilia patients a one-time dose of new clotting genes instead of a lifetime of multiple injections of recombinant factor IX that until very recently had to be given several times a week. A new FDA-approved hemophilia treatment lasts longer than a few days but patients still require injections at least once or twice a month indefinitely. This new gene therapy approach, like other gene therapy approaches, would involve a single injection and could … Continue reading →

Using mutant protein, CHOP hematologist safely removes unwanted antibodies, reverses hemophilia in dog model of bleeding disease Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. In addition, the "turbocharged" clotting factor protein eliminated pre-existing antibodies that often weaken conventional treatments for people with hemophilia. "Our findings may provide a new approach to gene therapy for hemophilia and perhaps other genetic diseases that have similar complications from inhibiting antibodies," said the study leader, Valder R. Arruda, M.D., Ph.D., a hematology researcher at The Children's Hospital of Philadelphia (CHOP). Arruda and colleagues published their animal study results in the print edition of Blood on March 5. Hemophilia is an inherited bleeding disorder that famously affected European royal families descended from Queen Victoria. Most commonly occurring in two types, hemophilia A and hemophilia B, the disease impairs the blood's ability to clot, sometimes fatally. When not fatal, severe hemophilia causes painful, often disabling internal bleeding and joint damage. Doctors treat hemophilia with frequent intravenous infusions of blood clotting proteins called clotting factors, but these treatments are expensive and time-consuming. Moreover, some … Continue reading →

FRESNO, Calif. (KFSN) -- Six-year-old Andy Moorhead is learning how to read. But instead of using his eyes, he's using his fingers. Andy told ABC30, "Well, I read the letters with my fingers." Andy is blind. Andy's Mother, Heather Ingram-Moorhead explained, "He was around nine months, and we started to notice his eyes were twitching." Andy has leber congenital amaurosis, or LCA. It's the most common type of childhood blindness and is caused by genetic mutations. "It is just very hard. It's taken us a while to really understand the condition and do everything to help Andy," Heather told ABC30. Andy's whole family is hands-on. Even his sister Valerie gives him guidance. But despite their efforts, his mom says gene therapy is their only hope. University of Florida scientist Shannon E. Boye, PhD, is using a $900,000 grant to perfect a gene therapy that could restore vision. "It's not an attempt just to slow the progression of the disease. It's actually an attempt to halt the progression and make the patient better by delivering them the gene they don't have," Boye told ABC30. Boye says the therapy has worked in animals. "We're able to show, via what's called an electra … Continue reading →

1. Introduction This chapter provides an overview of the main current applications of gene therapy for chronic pain in what concerns animal studies and putative clinical applications. The value of gene therapy in unravelling neuronal brain circuits involved in pain modulation is also analysed. After alerting to the huge socioeconomic impact of chronic pain in modern societies and justifying the need to develop new avenues in pain management, we review the most common animal studies using gene therapy, which consisted on deliveries of replication-defective viral vectors at the periphery with the aim to block nociceptive transmission at the spinal cord. Departing from the data of these animal studies, we present the latest results of clinical trials using gene therapy for pain management in cancer patients. The animal studies dealing with gene delivery in pain control centres of the brain are analysed in what concerns their complexity and interest in unravelling the neurobiological mechanisms of descending pain modulation. The chapter will finish by analysing possible futures of gene therapy for chronic pain management based on the development of vectors which are safer and more specific for the different types of chronic pain. Pain is not easy to define since it is … Continue reading →

An effective vaccine for HIV has eluded researchers for several decades, due to the pathogen's infamous shape-shifting abilities. Even though researchers have identified certain broadly neutralizing antibodies that can conquer multiple strains of the human immunodeficiency virus, many strains of rapidly mutating HIV remain resistant to the these super antibodies. In recent years however,researches have proposed a new method of battling the virus that involves gene therapy. Instead of using a vaccine to stimulate the body's own immune system, so that it produces HIV antibodies, scientists are bypassing the immune system entirely. In experiments involving rats and monkeys, the researchers have used non-life-threatening viruses to alter the animals' genome so that its cells produce designer molecules capable of neutralizing HIV. In a paper published Wednesday in the journal Nature, a team of researchers said they had used the technique to protect rhesus macaques from repeated intravenous injections of a SHIV, a combination of simian immunodeficiency virus and humanimmunodeficiency virus. The technique, researchers said, "can function like an effective HIV-1 vaccine." (HIV-1 is the main family of the virus, and accounts for most infections worldwide.) When HIV enters the body, it attacks specific immune cells. As the virus copies itself over … Continue reading →

An effective vaccine for HIV has eluded researchers for several decades, due to the pathogen's infamous shape-shifting abilities. Even though researchers have identified certain broadly neutralizing antibodies that can conquer multiple strains of the human immunodeficiency virus, many strains of rapidly mutating HIV remain resistant to the these super antibodies. In recent years however,researches have proposed a new method of battling the virus that involves gene therapy. Instead of using a vaccine to stimulate the body's own immune system, so that it produces HIV antibodies, scientists are bypassing the immune system entirely. In experiments involving rats and monkeys, the researchers have used non-life-threatening viruses to alter the animals' genome so that its cells produce designer molecules capable of neutralizing HIV. In a paper published Wednesday in the journal Nature, a team of researchers said they had used the technique to protect rhesus macaques from repeated intravenous injections of a SHIV, a combination of simian immunodeficiency virus and humanimmunodeficiency virus. The technique, researchers said, "can function like an effective HIV-1 vaccine." (HIV-1 is the main family of the virus, and accounts for most infections worldwide.) When HIV enters the body, it attacks specific immune cells. As the virus copies itself over … Continue reading →