A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

LEXINGTON, Ky.--(BUSINESS WIRE)--Demeetra AgBio, a genome engineering company enabling research and biomanufacturing through advanced gene-editing technologies and engineered CHO host cells, announced ...

Creative Biolabs is accelerating gene therapy research globally with specialized expertise. SHIRLEY, NY, UNITED STATES, ...

Progeria, an ultrarare and fatal genetic disease characterized by rapid aging, can affect children and young adults.

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, ...

Gene editing refers to the precise alteration of an organism's DNA sequence at a specific locus. In cancer treatment, this technology is leveraged to modify the genetic makeup of cancer cells, either ...

From an early age, Allison Keeler ’05 (biotechnology) always knew she wanted to be a scientist. As an adult, that dream has come true as she is an assistant professor in the Department of Pediatrics ...

Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy—the transplantation of living cells—and gene therapy—the use of ...

A Carlsbad family is sharing their son’s story after the 5-year-old was diagnosed with a rare genetic disorder and began a ...