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

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 ...

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

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

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

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 ...

Research led by Children's Hospital of Fudan University in China has found that a gene called pancreatic progenitor cell ...

A surprising mix of inherited and de novo mutations in 60 genes contribute to 10 percent of CHD cases. Many of these same ...

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 ...

AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 ...

Researchers have developed 'tomoseqr' -- a new software tool that enables easy estimation of the three-dimensional (3D) spatial distribution of gene expression. Tomoseqr is free to use and has been ...