Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...

Market OverviewThe global cell and gene therapy market is undergoing rapid and transformative growth, reflecting its increasing importance in modern healthcare.The market is projected to expand at a ...

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

Studies of Hunter and Hurler syndromes on hold ...

At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...

Researchers have developed a gene therapy that significantly slowed motor function loss in preclinical models of amyotrophic lateral sclerosis (ALS), offering new hope for treating the devastating ...

Genetic errors can cause many different diseases, and scientists have been looking for methods to fix those errors for decades. Although many gene therapies have aimed to correct errors in individual ...

The cochlea is the spiral-shaped structure within the inner ear responsible for our sense of hearing. To fully understand hearing functions and open the door to new hearing loss treatments, scientists ...

Eli Lilly plans to expand its hearing loss therapy pipeline by collaborating with Seamless Therapeutics to apply the company’s next-generation gene-editing approach, based on programmable recombinases ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

A protective APOE gene variant is enriched in Super Agers—people over 80 who think like 50‑year‑olds—cutting Alzheimer’s risk ...