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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
New gene therapy shows promising results for treating neurodegenerative disorders

New gene therapy shows promising results for treating neurodegenerative disorders

A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold. [More]
Researchers test new approach to treat metabolic diseases without organ transplant

Researchers test new approach to treat metabolic diseases without organ transplant

With a shortage of donor organs, Mayo Clinic is exploring therapeutic strategies for patients with debilitating liver diseases. Researchers are testing a new approach to correct metabolic disorders without a whole organ transplant. Their findings appear in Science Translational Medicine. [More]
Researchers find potential way for delivering gene therapy to treat eye diseases

Researchers find potential way for delivering gene therapy to treat eye diseases

Eye diseases such as diabetic retinopathy and age-related macular degeneration are among the leading causes of irreversible vision loss and blindness worldwide. Currently, gene therapy can be administered to treat these conditions -- but this requires an injection. [More]
Study reveals long-term safety of AAV2-neurturin gene therapy in patients with Parkinson's disease

Study reveals long-term safety of AAV2-neurturin gene therapy in patients with Parkinson's disease

New safety data from a study of patients with advanced Parkinson's disease five years after gene transfer-mediated delivery of the neuroprotective factor neurturin directly to patients' brains reveal no serious adverse events related to the treatment. [More]
Researchers discover three new risk genes for ALS

Researchers discover three new risk genes for ALS

Published today in Nature Genetics, the study reveals three new risk genes for ALS and one of these - C21orf2 - increases an individual's risk of developing the dis-ease by 65 per cent. [More]
Adhesive patch can deliver triple combination therapy to tumor sites

Adhesive patch can deliver triple combination therapy to tumor sites

Approximately one in 20 people will develop colorectal cancer in their lifetime, making it the third-most prevalent form of the disease in the U.S. In Europe, it is the second-most common form of cancer. [More]
New virus-based method opens wide range of options to treat various diseases

New virus-based method opens wide range of options to treat various diseases

The ability to switch disease-causing genes on and off remains a dream for many physicians, research scientists and patients. [More]
AMSBIO launches new CRISPR gRNA Lentivector Cloning Kits

AMSBIO launches new CRISPR gRNA Lentivector Cloning Kits

Anglo-American life science firm AMSBIO has introduced new CRISPR gRNA Lentivector Cloning Kits that provide scientists with a targeted and precise genomic gene editing methodology. [More]
Scientists develop new way to resurface arthritic hip joint

Scientists develop new way to resurface arthritic hip joint

With a goal of treating worn, arthritic hips without extensive surgery to replace them, scientists have programmed stem cells to grow new cartilage on a 3-D template shaped like the ball of a hip joint. [More]
The Wistar Institute and partners receive HIV cure research grant to test novel immunotherapies

The Wistar Institute and partners receive HIV cure research grant to test novel immunotherapies

The Wistar Institute is pleased to announce that the National Institutes of Health has awarded a nearly $23 million Martin Delaney Collaboratories for HIV Cure Research grant to the BEAT-HIV: Delaney Collaboratory to Cure HIV-1 Infection by Combination Immunotherapy, a consortium of top HIV researchers led by co-principal investigators Luis J. Montaner, D.V.M., D.Phil., director of the HIV-1 Immunopathogenesis Laboratory at The Wistar Institute Vaccine Center, and James L. Riley, Ph.D., research associate professor at the Perelman School of Medicine at the University of Pennsylvania. [More]
Scientists reveal how manipulating novel target in the brain could lead to new therapies for depression

Scientists reveal how manipulating novel target in the brain could lead to new therapies for depression

Northwestern Medicine scientists have shown how manipulating a novel target in the brain using gene therapy could lead to new treatments for depression. [More]
Exosome research and quality control at Cell Guidance Systems use the ZetaView

Exosome research and quality control at Cell Guidance Systems use the ZetaView

Cell Guidance Systems (CellGS) provides innovative life science research tools and services. Their goal is to identify valuable innovations from research groups around the world and develop products that address unmet needs, providing new tools that expand the possibilities of life science research. [More]
New book provides more insight into diagnosis and research of hereditary hearing loss

New book provides more insight into diagnosis and research of hereditary hearing loss

Genetics of Deafness offers a journey through areas crucial for understanding the causes and effects of hearing loss. [More]
New lung-targeted gene therapy shows promise in improving treatment for emphysema

New lung-targeted gene therapy shows promise in improving treatment for emphysema

Researchers have developed a new strategy using lung-targeted gene therapy that may lead to improved treatments for inherited diseases including emphysema. [More]
LouLou Foundation, ODC announce 11 new grants for research on CDKL5 deficiency

LouLou Foundation, ODC announce 11 new grants for research on CDKL5 deficiency

LouLou Foundation and the Orphan Disease Center (ODC) in the Perelman School of Medicine at the University of Pennsylvania are pleased to announce the award of 11 new research grants into CDKL5 deficiency - a rare X-chromosome-linked genetic disorder that causes severe neuro-developmental impairment and early-onset, difficult-to-control seizures. [More]
New AAV 3.0 program to create next-generation viral vectors for better treatment of inherited diseases

New AAV 3.0 program to create next-generation viral vectors for better treatment of inherited diseases

The Perelman School of Medicine at the University of Pennsylvania has launched a new program, called AAV 3.0, to create new viral vectors to find quicker and better treatments for an array of diseases. James M. Wilson, MD, PhD, a professor of Medicine and director of the Orphan Disease Center, will lead an interdisciplinary team of over 30 scientists to create this new technology platform with support provided by the University of Pennsylvania Health System. [More]
Researchers reveal novel mechanism by which viral protein VII suppresses immune alarm signals

Researchers reveal novel mechanism by which viral protein VII suppresses immune alarm signals

Viruses must avoid a host's immune system to establish successful infections—and scientists have discovered another tool that viruses use to frustrate host defenses. [More]
NYU dentists receive grant to test whether non-viral gene delivery can effectively treat oral cancer pain

NYU dentists receive grant to test whether non-viral gene delivery can effectively treat oral cancer pain

The National Institute of Dental and Craniofacial Research, part of the National Institutes of Health, has awarded Drs. Brian Schmidt and Seiichi Yamano a $1.2M (3-year) grant to test whether their non-viral gene delivery method can effectively and safely treat oral cancer pain. [More]
New book highlights advances, challenges in development of nanotheranostics for personalized medicine

New book highlights advances, challenges in development of nanotheranostics for personalized medicine

The future of medicine is now dawning upon us.Nanomedicines demonstrate the capability to enhance drug properties by offering protection from degradation, enabling controlled release and biodistribution and increasing bioavailability. [More]
Gamma-ray irradiation improves safety of stem cell transplantation in Parkinson’s disease patients

Gamma-ray irradiation improves safety of stem cell transplantation in Parkinson’s disease patients

Replacing dopamine-producing cells in the brain represents a promising therapeutic approach in Parkinson's disease, and a new study shows how post-transplantation gamma-ray irradiation can reduce the risk of tumor formation. [More]
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