Gene Therapy News
June 25, 2019

Top Headlines

Researchers Use Gene Editing With CRISPR to Treat Lethal Lung Diseases Before Birth

Using CRISPR gene editing, researchers have thwarted a lethal lung disease in an animal model in which a harmful mutation causes death within hours ... read more

Gene Therapy Restores Immunity in Infants With Rare Immunodeficiency Disease

A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening ... read more

Gene Therapy Durably Reverses Congenital Deafness in Mice

Scientists have managed to restore hearing in an adult mouse model of DFNB9 deafness -- a hearing disorder that represents one of the most frequent cases of congenital genetic deafness. Individuals ... read more

Guided by CRISPR, Prenatal Gene Editing Shows Proof-of-Concept in Treating Disease Before Birth

For the first time, scientists have performed prenatal gene editing to prevent a lethal metabolic disorder in laboratory animals, offering the ... read more
Latest Headlines
updated 12:18am EDT

Earlier Headlines

Brighter Possibilities for Treating Blindness

Advances in preclinical research are now being translated into innovative clinical solutions for blindness, a review ... read more

Germline Gene Therapy: Safety

An internationally known embryologist and his son make the case for using gene-editing tools to prevent inherited disease, in a new editorial. The authors push back against recent calls for a ... read more

New Role in Spatial Chromosome Organization Identified for Often Mutated Cancer Protein

New research sheds light on the function of the ARID1A protein, encoded by a gene that is among the most frequently mutated across human ... read more

A Specific Gene Could Play a Major Role in Reducing Brain Swelling After Stroke

Inflammation gone awry in the brain due to stroke, head injury or infection causes damage; in a lab model of stroke, a particular gene tamped down ... read more

DNA Methylation Levels Linked to Obesity

A multidisciplinary study has linked DNA methylation levels to the development of the metabolic disease associated with obesity. A multidisciplinary work that has been carried out by biologists, ... read more

Making 'Sense' of the 'Cart Before the Horse' in Mammalian Cells

Researchers show that antisense RNA can mediated the formation of fusion ... read more

Gene Therapy Promotes Nerve Regeneration

Researchers have shown that treatment using gene therapy leads to a faster recovery after nerve damage. By combining a surgical repair procedure with gene therapy, the survival of nerve cells and ... read more

Gene Therapy Blocks Peripheral Nerve Damage in Mice

Scientists have developed a gene therapy that blocks axonal degeneration, preventing axon destruction in mice and suggesting a therapeutic strategy that could help prevent the loss of peripheral ... read more

Researchers Overcome Hurdle in CRISPR Gene Editing for Muscular Dystrophy

The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions. A ... read more

Eliminating the Latent Reservoir of HIV

A new study suggests that a genetic switch that causes latent HIV inside cells to begin to replicate can be manipulated to completely eradicate the virus from the human body. Cells harboring latent ... read more

Gene Editing Possible for Kidney Disease

For the first time scientists have identified how to halt kidney disease in a life-limiting genetic condition, which may pave the way for personalised treatment in the ... read more

Gene Signature Discovery May Predict Response to Immune Therapy

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

Realizing the Potential of Gene Therapy for Neurological Disorders

Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders. Scientists have successfully used gene therapy to slow the ... read more

New Study Offers Hope for Patients Suffering from a Rare Form of Blindness

A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness. This hyper-targeted approach offers hope to individuals living with ... read more

CRISPR Gene Editing Will Find Applications in Plastic and Reconstructive Surgery

The CRISPR genome editing technique promises to be a 'transformative leap' in genetic engineering and therapy, affecting almost every area of medicine. That includes plastic surgery, with ... read more

Preventing Sudden Cardiac Death With Genome Editing

Gene editing successfully prevented sudden cardiac death in a mouse model of inherited cardiac arrhythmia ... read more

Normal Function of ALS and Dementia Linked Gene Determined for the First Time

The normal function of a gene associated with the neurodegenerative diseases amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) has been determined for the first ... read more

Making Gene Therapy Delivery Safer and More Efficient

Viral vectors used to deliver gene therapies undergo spontaneous changes during manufacturing which affects their structure and function. As gene therapy approaches become more common for treating ... read more

Novel Immune Syndrome Described

Researchers have discovered a new human immunodeficiency syndrome in two patients on separate continents. The study reveals that a mutation in a gene called IKBKB disrupts the immune system, leading ... read more

This RNA-Based Technique Could Make Gene Therapy More Effective

Biological engineers have devised a way to regulate the expression of messenger RNA once it gets into cells, giving them more precise control over gene therapy treatments for cancer and other ... read more

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