Why are some people unable to hear from birth, even though their inner ear appears intact? One possible cause lies in the so-called OTOF gene. It plays a central role in transmitting sound signals ...

CRISPR-based modification of haematopoietic stem cells has enabled durable, boostable antibody production in mouse models ...

In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification ...

Researchers have determined whether a specific chemical modification of a protein that packages the genome called a histone affects gene activity and cell proliferation. Researchers have determined ...

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have ...

A committee-supported project or activity that has been completed and for which output dissemination has begun. Its committee has been disbanded and closeout procedures are underway. An ad hoc ...

Genome editing is a powerful biotechnology tool that allows scientists to make precise changes to an organism's DNA. It involves the use of specialized enzymes, known as engineered nucleases or ...

Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including our reproductive cells. While most of these defects have no or only minor ...

Philadelphia, July 28, 2023—In a step forward in the development of genetic medicines, researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University ...

An ad hoc committee appointed by the National Academies to explore heritable genetic modifications of food animals. This committee will address methods for heritable genetic modifications and identify ...