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Gene Therapy News
August 2, 2023

Top Headlines

Researchers Develop 'in Vivo' RNA-Based Gene Editing Model for Blood Disorders

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the ...

Researchers Pinpoint Protein Tied to Drug Resistance in Patients With Lung Cancer

Cancer therapies that target specific genetic abnormalities in tumors have revolutionized treatment possibilities over the past two decades. While quality of life and survival are improved with targeted therapies, relapse is common due to the ...

The Drug Fasudil Is Found to Reverse Key Symptoms of Schizophrenia in Mice

Researchers used the drug fasudil to restore neurons and improve methamphetamine-induced cognitive dysfunction in a mouse model of ...

Silicon Nanochip Could Treat Traumatic Muscle Loss

技术,可以改变皮肤组织变为血vessels and nerve cells also shows promise as a treatment for traumatic muscle ...
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Latest Headlines
updated 2:31pm EDT

Earlier Headlines

Peering Into Ocular Waste Recycling

Researchers uncover a mechanism in a retinal transporter protein that causes blindness, could lead to targeted ...

Liver Cancer Study Encourages Caution With Certain Gene Therapies

Research has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases. ...

New Gene Therapy Shows Promise for Treating Eye Condition Affecting Millions Across the Globe

Researchers have developed a new gene therapy approach that shows promise for treating the dry form of Age Related Macular Degeneration (AMD) -- a progressive eye disease that affects up to 10% of ...

Mouse Study Shows Gene Therapy May Correct Creatine Deficiency Disorder

A new study in mice finds that a gene therapy appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements ...

Gene Therapy Could Treat Pitt-Hopkins Syndrome, Proof-of-Concept Study Suggests

Scientists reported on an experimental gene-therapy-like technique to restore the normal activity of the TCCF4 gene deficient in individuals with Pitt-Hopkins ...

Establishment of a Pancreatic Cancer Animal Model Using the Pancreas-Targeted Hydrodynamic Gene Delivery Method

Pancreatic cancer has a significantly poor prognosis; therefore, the development of effective treatments is an unmet clinical need. The major drawback in this field was the lack of useful model ...

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DNA Discovery Reveals a Critical 'Accordion Effect' for Switching Off Genes

Researchers have revealed how an 'accordion effect' is critical to switching off genes, in a study that transforms the fundamentals of what we know about gene silencing. The finding expands ...

Novel Nuclear microRNA Is Being Developed for the Treatment of Cardiovascular Disease

一种新的血管生成微rna药物可以是一个新的option for the treatment of ischemic cardiovascular disease, according to a new study. In the study, the researchers describe a novel nuclear acting ...

An Approach to Treating a Severe Congenital Myopathy

The diagnosis is rare, but devastating -- children with congenital muscle disorders often never learn to walk. Until now, there was no chance of recovery, but researchers are now presenting a ...

How a Two-Faced Molecule Can Silence Problematic Genes

Researchers have developed a technology, heteroduplex oligonucleotide (HDO), that silences certain genes whose high expression levels fuel disease. Adding a specific molecular tag allowed them to ...

A Possible Cure for Sickle Cell?

Sickle cell anemia is an inherited blood disorder where red blood cells become sickle/crescent shaped. It causes frequent infections, swelling in the hands and legs, pain, severe tiredness and ...

My Heart Will Go On: Patient-Derived Heart Cells Mimic Disease in Vitro

Researchers have found that induced pluripotent stem cell--derived cardiomyocytes from a patient with arrhythmogenic cardiomyopathy recapitulate the reduced contractility and impaired desmosome ...

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Calcium: Important Not Just for Your Bones but Also for Your Heart

Researchers found a previously unknown gene mutation that can cause an incurable heart condition called dilated cardiomyopathy. This gene, BAC5, is important for the movement of calcium ions in the ...

Epilepsy Research Boosts Case for New Gene Therapy for Dravet Syndrome

New research suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the ...

New Gene Therapy Approach Offers a Potential Long-Term Treatment for Limb-Girdle Muscular Dystrophy 2B

Medical experts have developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this ...

Experimental Gene Therapy Reverses Sickle Cell Disease for Years

An experimental gene therapy for sickle cell disease restored blood cells to their normal shape and eliminated severe pain crises for years after treatment, a multicenter study has ...

Gene Network Changes Associated With Cancer Onset and Progression Identify New Treatment Targets

Researchers have identified novel changes in gene network interactions associated with cancer that may lead to new treatment targets for chemotherapy. Their work shows that more than 90 percent of ...

CRISPR/Cas9 Gene Editing Boosts Effectiveness of Ultrasound Cancer Therapy

Sonodynamic therapy uses ultrasound in combination with drugs to release harmful reactive oxygen species (ROS) at the site of a tumor. However, the treatment isn't very effective because cancer ...

ALS Therapy Should Target Brain, Not Just Spine

The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), scientists have discovered. This flips a long-standing belief the disease starts in the spinal motor neurons and any ...

Novel Gene Therapy Platform Speeds Search for Ways to Cure Blindness

一种新型计算平台标识top-performing viral vectors that could deliver gene therapies to the retina with maximum efficiency and ...

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