Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patient’s cells. In a finding with implications for one ...

Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and accurate ...

Scientists improved adenine base editing in mitochondrial DNA using engineered transcription activator-like effector-linked deaminases (TALEDs). The engineered TALEDs minimize off-target gene edits, ...

Early results from a study of newborn screening methods show that DNA analysis picks up many more preventable or treatable serious health conditions than standard newborn screening and is favored by ...

Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same ...

Early results from 4,000 babies show that genome sequencing picks up many more serious health conditions than standard newborn screening and is favored by most parents. Early results from a study of ...

One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid increases in genetic technology and testing. Even when a person does have ...

Much as ChatGPT generates poetry, a new A.I. system devises blueprints for microscopic mechanisms that can edit your DNA. The physical structure of OpenCRISPR-1, a gene editor created by A.I.

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid increases in genetic technology and testing. Even when a person does have ...