Researchers say the CRISPR-based technique used could eventually be employed to treat more people with rare genetic diseases ...

Durham-based Atsena Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for ATSN-201, its gene therapy product for the ...

With grim prognosis hanging overhead, doctors and scientists at universities and institutions across the U.S. worked ...

Evidence is mounting that clinicians can treat serious genetic disorders prenatally by injecting medicine into the amniotic fluid, thus preventing damage that begins in utero.

Researchers at Children’s Hospital of Philadelphia (CHOP) have developed advanced generations of adeno-associated viral (AAV) ...

For Uditi’s parents, reading about use of CRISPR tech, or ‘genetic scissors’, to treat baby KJ, diagnosed with severe genetic ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene-editing treatment made just for him. Researchers described the case in a new study, say ...

Mike McCune, MD, PhD, head of the HIV Frontiers Program at the Gates Foundation, delivered an upbeat message at the ASGCT annual conference in New Orleans last week, showcasing the progress being made ...

Autoimmunity BioSolutions (ABS), a biotechnology company pioneering targeted, immuno-corrective therapy for a highly ...

In the Phase 2 study of patients with chronic kidney disease, the highest dose of the drug lowered by 86% the levels of ...

In an era that FDA Commissioner Marty Makary, MD, MPH, calls an "epidemic of distrust" toward health institutions, previously ...

Isaacs says that the positive correlation he and his colleagues found between the mutated MMS22L gene and prostate cancer ...