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 ...

SCD gene therapy approaches using CRISPR, gene therapy, and base editing show different stem cell outcomes in a mouse study.

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 ...

Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or inactivating genes. Precisely fixing disease-causing mutations is far more ...

Chitra Kannabiran, PhD, Kallam Anji Reddy Molecular Genetics Laboratory, Hyderabad Eye Research Foundation, Champalimaud Translational Centre, LV Prasad Eye Institute, Hyderabad 500 034, India ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

BUFFALO, N.Y. — A viral gene therapy developed by University at Buffalo researchers has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating ...

Each year hundreds of children are born legally blind because of a genetic disorder that degrades their retinas. Though some can distinguish between light and dark early on, a defect in their AIPL1 ...