Genetics

New way forward for gene therapy

A team of scientists from Children's Medical Research Institute (CMRI) in Sydney have developed a new way to improve targeting of specific organs and tissue types in gene therapy—making this innovative gene delivery technology ...

Neuroscience

Scientists gain ground on rare congenital neurological disorder

Two recent discoveries co-led by scientists at Cedars-Sinai may help lead to new ways to treat patients with Allan-Herndon-Dudley syndrome (AHDS), a brain development disorder that causes severe intellectual disability ...

Diseases, Conditions, Syndromes

The cost of living with sickle cell disease

In a study published today in Blood Advances, researchers found that privately insured individuals with sickle cell disease (SCD) spend approximately $1.7 million on disease-related medical expenses over their lifetime. These ...

Medical research

Defining the role of HDAC6 in regulating heart stiffness

Researchers at the University of Colorado School of Medicine have discovered an enzyme that regulates heart stiffness, setting the stage for developing novel treatments for heart failure.

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Gene therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.

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