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Gene Therapy News
December 12, 2018

Latest Headlines
updated 7:18am EST

More Gene Therapy News
December 12, 2018

Gene Signature Discovery May Predict Response to Immune Therapy

Nov. 8, 2018 — Scientists have discovered a gene signature biomarker that may predict which patients will respond -- or not -- to immune ... read more

Disease Causing Mutation Found in French-Canadians

Oct. 1, 2018 — A team of scientists has discovered the first French-Canadian founder mutation gene linked to synucleinopathies, a group of neurodegenerative diseases that includes Parkinson's disease (PD), ... read more

Sep. 17, 2018 — A promising gene therapy for visually impaired sheep is now safe for human ... read more

How Exercise Generates New Neurons, Improves Cognition in Alzheimer's Mouse

Sep. 6, 2018 — A study finds that neurogenesis -- inducing the production of new neurons -- in the brain structure in which memories are encoded can improve cognitive function in a mouse model of Alzheimer's ... read more

Scientists Engineer Way to Prevent Immune Response to Gene Therapy in Mice

Sep. 3, 2018 — Researchers have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which the ... read more

New Genetic Marker Could Help Diagnose Aggressive Prostate Cancer

Aug. 30, 2018 — A new link has been found between certain genetic mutations, the aggressiveness of prostate cancer, risk of developing the disease and poorer survival rates of ... read more

Gene Therapy Vectors Carrying the Telomerase Gene Do Not Increase the Risk of Cancer

Aug. 20, 2018 — Researchers have shown in a new study that the gene therapy with telomerase that they have developed, and which has proven to be effective in mice against diseases caused by excessive telomere ... read more

Aug. 14, 2018 — An aquarium fish that senses the Earth's magnetic field as it swims could help unlock how the human brain works and how diseases such as Parkinson's and other neurological disorders ... read more

Amish Nemaline Myopathy Natural History Study Finds Promise for Gene Therapy Treatment

Aug. 13, 2018 — A new comprehensive natural history study about Amish nemaline myopathy (ANM) in the Old Order Amish population focuses on the promise of gene therapy for this lethal disorder. Amish nemaline ... read more

Aug. 2, 2018 — The fight against cystic fibrosis has taken a major step forward, with new research showing that cells causing the debilitating genetic disorder could be successfully replaced with healthy ... read more

Gene Therapy: Better Adenine Base Editing System

July 31, 2018 — Scientists have developed and improved the ABE system in mouse and rat strains, which has great implications for human genetic disorders and gene ... read more

July 26, 2018 — Researchers delayed signs of amyotrophic lateral sclerosis (ALS) in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1 (SOD1). The results ... read more

Mutated Gene Is Likely Cause of 3-Year-Old Girl's Severe Disorder

July 23, 2018 — A young girl with severe neurological symptoms finally has a diagnosis, following the discovery of a genetic mutation that likely caused the girl to experience a cascade of symptoms. Like a train ... read more

Genome Damage from CRISPR/Cas9 Gene Editing Higher Than Thought

July 19, 2018 — Scientists have discovered that CRISPR/Cas9 gene editing can cause greater genetic damage in cells than was previously thought. This has safety implications for future gene therapies using ... read more

Genome Editing Reduces Cholesterol in Large Animal Model, Laying Human Trial Groundwork

July 9, 2018 — Using genome editing to inactivate a protein called PCSK9 effectively reduced cholesterol levels in rhesus macaques. This is the first demonstration of a clinically relevant reduction of gene ... read more

Gene Therapy Shown to Cure Type 2 Diabetes and Obesity in Mice, Researchers Report

July 9, 2018 — A single administration of a therapeutic vector in mouse models cures type 2 diabetes and obesity in the absence of long-term side effects, researchers report. In healthy mice, the therapy prevents ... read more

July 5, 2018 — Research has shown, in mice, that genetic material can be delivered to damaged cells in the kidneys, a key step toward developing gene therapy to treat chronic kidney disease. The potentially fatal ... read more

New Nuclear Medicine Technique Could Help Tackle Brain Disease

June 25, 2018 — A new molecular imaging method can monitor the success of gene therapy in all areas of the brain, potentially allowing physicians to more effectively tackle brain conditions such as Parkinson's ... read more

Using Gene Silencing to Alleviate Common Ataxia

June 20, 2018 — In what researchers are calling a game changer for future ataxia treatments, a new study showed the ability to turn down the disease progression of the most common dominantly inherited ataxia, ... read more

Genome-Editing Tool Could Increase Cancer Risk

June 11, 2018 — Therapeutic use of gene editing with the so-called CRISPR-Cas9 technique may inadvertently increase the risk of cancer, according to a new study. Researchers say that more studies are required in ... read more

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