The growing occurrence of genetic disorders like sickle cell anemia, cystic fibrosis, and muscular dystrophy is fueling the demand for gene therapy and genome editing solutions, as these approaches ...

10, 2024 — In a single IV injection, a gene therapy ... a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough ...

This event will focus on the latest progress in precision genome editing, novel delivery vectors ... laboratory research into clinical-grade cell and gene therapies. Explore how innovations in cell ...

In December 2023, through the development efforts of CRISPR Therapeutics and Vertex Pharmaceuticals, their decades-long endeavor reached fruition in the form of a new treatment, CASGEVY, approved by ...

A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.

Mitochondrial deoxyribonucleic acid (mtDNA) is essential for cellular energy production and overall cell function.

Learn how CASGEVY, the first NHS-approved CRISPR gene therapy, treats sickle cell disease through a revolutionary genetic ...

This study presents an enhanced nuclease prime editor (uPEn) that significantly improves gene knock-in and knockout ...

Imagine a breakthrough in cancer treatment where only malignant cells are targeted, sparing healthy host cells; or patients with abnormal protein synthesis are treated to produce a healthy protein.

PRGN-2012—which Precigen has begun to call by its generic name of zopapogene imadenovec—is a gene therapy designed to elicit immune responses directed against cells infected with human papillomavirus ...

Editas Medicine, Inc.’s EDIT share price has surged by 16.48%, which has investors questioning if this is right time to sell.

At the Envision Summit 2025 in San Juan, Puerto Rico, Mike Farkas, MD, discussed gene therapy research for retinal ...