The goal of genome editing technology is to develop strategies for the correction of those mutations that are responsible of ...

The U.S. Food and Drug Administration approved Soleno Therapeutics' drug to treat a rare genetic disorder on Wednesday, ...

What Are Inherited Metabolic Disorders? Inherited metabolic disorders are genetic conditions that result in metabolism problems. Most people with inherited metabolic disorders have a defective ...

Genetic engineering in non-human primates has long been limited by the need for virus-based gene delivery methods. Recently, ...

Affecting 1 in 500 people, hypertrophic cardiomyopathy is a condition in which the walls of the left ventricle, the heart's ...

Congenital heart disease (CHD) is one of the most common birth defects, but the full extent of its genetic underpinnings has ...

Base editing corrected a mutation that causes macular degeneration, highlighting the potential of gene therapy to treat ...

A genetic mutation in horses that would typically halt protein production has become a molecular asset. Researchers at Johns ...

Genetic engineering in non-human primates has long been limited by the need for virus-based gene delivery methods. Recently, ...

Japanese researchers used a nonviral piggyBac system to genetically modify cynomolgus monkeys, enabling more accurate disease ...

Advances in the gene-editing technology known as CRISPR-Cas9 over the past 15 years have yielded important new insights into the roles that specific genes play in many diseases. But to date this ...

Single IV administration of tau silencing gene therapy VY1706 significantly reduced tau mRNA and protein levels, with broad brain distribution ...