Tiny Bubbles and a Bit of Gene Therapy Heal Major Bone Fractures in Pigs

It takes more than a cast and a little time to heal many broken bones. Whether it’s a soldier wounded in battle, a car accident victim, or an elderly person who has fallen, bone damage can be so extensive that the bones never heal properly, leaving people crippled or with other severe problems. Now, researchers have combined ultrasound, stem cells, and gene therapy to stimulate robust bone repair. So far the work has only been performed in animals. But it has already been so successful that it’s expected to move quickly toward human clinical trials. The new research “has huge clinical significance,” says David Kulber, who directs the Center for Plastic and Reconstructive Surgery at Cedars-Sinai Medical Center in Los Angeles, California, and who was not part of the study. “The technology of being able to stimulate bone growth is really remarkable.” It’s also one for which there is a glaring need. In the United States alone, some 100,000 people a year suffer from what is known as a nonunion fracture. In these cases, parts of a bone may be missing altogether or so badly splintered that the bone can’t be reassembled. In such cases, doctors typically graft other bone into the site. Ideally this bone comes from the same person—often taken from the pelvis, a painful procedure that compounds a person’s injuries and recovery time. When this isn’t possible, physicians will turn to cadavers for the extra bone. But this bone must be sterilized before it’s implanted, robbing it of proteins and other signaling molecules that encourage its regrowth once transplanted, and lessening the chances of a full recovery.
read more.-->

Gene Therapy to Strengthen Nerve-Muscle Connection Prevents ALS Progression in Mice

A gene therapy aimed at strengthening the connection between motor neurons and muscle prevented nerve degeneration and increased the life span of mice with amyotrophic lateral sclerosis (ALS), a study showed. Since researchers believe that neurodegeneration in ALS starts at the junction between nerves and muscle, the findings suggest that preventing the connection from breaking down could be a good way to stop the disease from progressing. The study, “DOK7 gene therapy enhances motor activity and life span in ALS model mice,”was published in the journal EMBO Molecular Medicine. Interestingly, the University of Tokyo researchers did not develop the gene therapy with ALS in mind. They were trying to prevent a disease called DOK7 myasthenia that is caused by mutations of the DOK7 gene.
read more.-->

Type 1 Diabetes Cured in Mice Using Gene Therapy

Researchers from the University of Texas Health Science Center in San Antonio have found a way to cure type 1 diabetes in mice. It is hoped that the novel technique - which boosts insulin secretion in the pancreas - will reach human clinical trials in the next 3 years. Study co-author Dr. Bruno Doiron, Ph.D., of the Division of Diabetes, and colleagues recently reported their findings in the journal Current Pharmaceutical Biotechnology. Type 1 diabetes is estimated to affect around 1.25 million children and adults in the United States. Onset of the condition is most common in childhood, but it can arise at any age.
read more.-->

Expensive Gene Therapy Receives its First Patient in a Commercial Treatment

A patient with an extremely rare immune disease has been treated with commercial gene therapy for the first time, GlaxoSmithKline, the company behind the therapy, told MIT Technology Review on Tuesday. The treatment come almost a year after the therapy was approved for sale in Europe. Known as Strimvelis, the therapy treats a rare inherited immune deficiency by fixing a problem within the patient’s DNA. Gene therapy has been used extensively in clinical trials but has had a slow start commercially. This is only the second commercial use of gene therapy, the first of which was with a drug called Glybera in 2015.
read more.-->

First Clear-cut Risk Genes for Tourette Disorder Revealed

Tourette disorder (also known as Tourette syndrome) afflicts as many as one person in a hundred worldwide with potentially disabling symptoms including involuntary motor and vocal tics. However, researchers have so far failed to determine the cause of the disorder, and treatments have only limited effectiveness, in part because the genetics underlying the disorder have remained largely a mystery. Now, as reported online May 3, 2017 in Neuron, a consortium of top researchers -- led by scientists at UC San Francisco, Rutgers University, Massachusetts General Hospital, the University of Florida, and Yale School of Medicine -- has made a significant advance, identifying the first "high-confidence" risk gene for Tourette disorder as well as three other probable risk genes. These findings are a step forward in understanding the biology of the disorder, the authors said, which will aid in the search for better treatments.
read more.-->

GenSight Biologics’ Gene Therapy Candidate May Help Vision Loss in LHON Patients

An investigational gene therapy, GS010, is showing some promise in helping people who recently began to lose their vision due to Leber’s hereditary optic neuropathy (LHON), according to data from an ongoing clinical trial. A presentation of the data, “Intravitreal rAAV2/2-ND4 (GS010): A gene therapy for vision loss in Leber’s Hereditary Optic Neuropathy (LHON) caused by the G11778A ND4 mitochondrial mutation,” is set for April 25 at the 2017 annual meeting of the American Association of Neurology (AAN), taking place through April 28 in Boston. It will be given by José-Alain Sahel, MD, co-founder of GenSight Biologics, the developer of GS010. The Paris-based company’s lead product candidate, GS010, was designed to be the first gene replacement therapy targeting LHON, a rare inherited mitochondrial disease caused by ND4 mitochondrial gene mutation. Mainly affecting adolescents and young adults, LHON is characterized by irreversible vision loss due to a degeneration of retinal ganglion cells. In more severe cases, this disease can lead to blindness.
read more.-->

Gene Therapy Trial Launched for X-linked Retinitis Pigmentosa

Researchers have injected their first patient with a virus engineered to remodel the gene responsible for X-linked retinitis pigmentosa (XLRP). “If successful, this gene therapy has the potential to transform the lives of many patients,” said David Fellows, chief executive officer, Nightstar, a gene therapy company in Oxford, according to an Oxford University press release. The injection took place as part of a multicenter open-label study designed to enrolat least 24 male patients in a 12-month trial of safety and tolerability. It is the first in the world to test a treatment for retinitis pigmentosa caused by the retinitis pigmentosa GTPase regulator (RPGR) gene, the press release said. One of the leading causes of blindness in young people, retinitis pigmentosa is currently untreatable and leads to a slow and irreversible loss of vision due to the loss of rods and cones.
read more.-->

Gene Therapy Shows Promise in Treating Deadly Brain Disorders

A novel ‘gene-silencing’ drug could be the key to treating two devastating neurological disorders, spinocerebellar ataxia type 2 (SCA2) and amyotrophic lateral sclerosis (ALS). SCA2 is an inherited disorder that inflicts damage on the brain’s cerebellum, causing patients to have issues with balance, coordination, walking and similar movements. ALS induces degeneration of nerve cells in the brain and spinal cord making patients gradually lose their ability to perform basic functions like move, speak, eat, or breathe. There are different factors that can initiate the onset of these diseases, but two new studies indicate the first signs of a possible treatment approach for both SCA2 and ALS.
read more.-->

Novel Gene Therapy Experiment Offers Hope for People with Certain Hearing Loss and Dizziness Disorder

In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene therapy was able to restore balance and hearing in genetically modified mice that mimic Usher Syndrome, a genetic condition in humans characterized by partial or total hearing loss, dizziness, and vision loss that worsens over time. The hearing loss and dizziness is caused by abnormalities of the inner ear. Dizziness and hearing loss are among the most common disabilities affecting humans and can be severe and debilitating. According to the National Health and Nutrition Examination Survey, more than 35% of U.S. adults aged 40 years and older have some degree of balance dysfunction, a major cause of falls in the elderly. According to the Centers for Disease Control, approximately one in three people in the United States between the ages of 65 and 74 has hearing loss, and nearly half of those older than 75 have difficulty hearing. Men are more likely to experience hearing loss than women.
read more.-->

Inovio Pharmaceutical’s DNA Vaccine will be Tested in Patients with HIV

Inovio Pharmaceuticals in Plymouth Meeting is collaborating with the University of California San Francisco, which received a $6.95 million grant from the National Institutes of Health, to test the biotech company's DNA-based vaccine to reduce or prevent the HIV virus. Inovio's immunotherapy, Pennvax GP, will be tested in HIV-positive patients to see if it generates killer T cells in the body's immune system to attack the HIV virus. Current antiviral drugs work well against HIV, "but people have to take these drugs every day for decades," said Steven Deeks, the grant's principal investigator and professor of medicine at the University of California, San Francisco. For many people around the world the drugs "are just not feasible" due to side effects or costs, he said. "We're trying to find a way to enable the immune system to do what antiretroviral drugs do, which is to prevent the virus from replicating and spreading in the person."
read more.-->