Gene Therapy News
October 29, 2020

Top Headlines

Restoration of Retinal and Visual Function Following Gene Therapy

A breakthrough study results in the restoration of retinal and visual functions of mice models suffering from inherited retinal ...

New Gene Correction Therapy for Duchenne Muscular Dystrophy

Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers have ...

Targeting a Blood Stem Cell Subset Shows Lasting, Therapeutically Relevant Gene Editing

Researchers have used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed with several blood disorders, ...

HIV Eliminated from the Genomes of Living Animals

Researchers have for the first time eliminated replication-competent HIV-1 DNA -- the virus responsible for AIDS -- from the genomes of living animals. The study marks a critical step toward the ...
Latest Headlines
updated 11:30am EDT

Earlier Headlines

Progress Toward a Treatment for Krabbe Disease

The inherited disease, which typically kills children before their second birthday, has no cure, but a new study in a canine model offers hope for an effective gene therapy with lasting ...

Simulations Show How to Make Gene Therapy More Effective

Diseases with a genetic cause could be treated by supplying a correct version of the faulty gene. However, in practice, delivering new genetic material to human cells is difficult. A promising method ...

Switching on a Key Cancer Gene Could Provide First Curative Treatment for Heart Disease

Researchers trying to turn off a gene that allows cancers to spread have made a surprising U-turn. By making the gene overactive and functional in the hearts of mice, they have triggered heart cell ...

New Research Suggests in-Womb Gene Correction

New research led by hearing scientists suggests an avenue to treat and prevent intractable genetic disorders before birth. Researchers, working with mice, injected a specially designed synthetic ...

Gene Therapy Reverses Heart Failure in Mouse Model of Barth Syndrome

Barth syndrome is a rare genetic disease in boys that can cause life-threatening heart failure and also weakens the skeletal muscles and the immune system. There is no specific treatment, but new ...

Cells Carrying Parkinson's Mutation Could Lead to New Model for Studying Disease

Parkinson's disease researchers have used gene-editing tools to introduce the disorder's most common genetic mutation into marmoset monkey stem cells and to successfully tamp down cellular ...

New CRISPR Base-Editing Technology Slows ALS Progression in Mice

With a new CRISPR gene-editing methodology, scientists have inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis -- a debilitating and fatal neurological ...

Novel Techniques for Mining Patented Gene Therapies Offer Promising Treatment Options

Scientists are working to gain a better understand of the growing number of worldwide patented innovations available for gene therapy ...

New Gene Therapy Method Improves Vision in Mice With Congenital Blindness

Mice born blind have shown significant improvement in vision after undergoing a new gene ...

Six Patients With Rare Blood Disease Are Doing Well After Gene Therapy Clinical Trial

Researchers have reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those ...

Mechanism for How Common Gene Therapy Vectors Enter Cells

Researchers have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types -- the most commonly used viral vectors for in vivo gene therapy. The researchers identified ...

Drug Profiling and Gene Scissors Open New Avenues in Immunotherapy

Researchers have discovered ways to boost CAR T-cell ...

Nanoparticles Deliver 'Suicide Gene' Therapy to Pediatric Brain Tumors Growing in Mice

Researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a ''suicide gene'' to pediatric brain tumor cells implanted in ...

Gene Therapy Shown to Offer Long-Term Benefits for People With Haemophilia A

A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the ...

New Technology Allows Control of Gene Therapy Doses

Scientists have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing. The feat offers gene therapy designers what may be the first viable ...

For CRISPR, Tweaking DNA Fragments Before Inserting Yields Highest Efficiency Rates Yet

Researchers have now achieved the highest reported rates of inserting genes into human cells with the CRISPR-Cas9 gene-editing system, a necessary step for harnessing CRISPR for clinical gene-therapy ...

High-Tech Method for Uniquely Targeted Gene Therapy Developed

Neuroscientists have developed new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe that the ...

Unique Case of Disease Resistance Reveals Possible Alzheimer's Treatment

Defying the odds, an individual at high risk for early-onset Alzheimer's disease remained dementia-free for many years beyond what was anticipated. A new study led researchers to suggest that a ...

New Gene Therapy for Epilepsy Provides on-Demand Release of Endogenous Substance

Scientists have developed a new therapeutic concept for the treatment of temporal lobe epilepsy. It represents a gene therapy capable of suppressing seizures at their site of origin on demand. Having ...

Discovery in Monkeys Could Lead to Treatment for Blindness-Causing Syndrome

A genetic mutation that leads to a rare, but devastating blindness-causing condition called Bardet-Biedl Syndrome has been discovered in monkeys for the first time. The finding offers a promising way ...

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