Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...

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 ...

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, ...

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 ...

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 ...

The power of artificial intelligence (AI) and advanced computing has made it possible to design genetic sequences encoding ...

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.

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 ...

Understanding human gene function in living organisms has long been hampered by fundamental differences between species. Although mice share most protein-coding genes with humans, their regulatory ...

A new study shows that long-read sequencing has the potential to improve the rate of diagnosis while reducing the time to diagnosis from years to days -- in a single test and at a much lower cost. One ...