Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

The Director of the San Raffaele-Telethon Institute for Gene Therapy Luigi Naldini reflects on what a recent Nature study reveals and about the future of in vivo gene transfer “Our study provides an ...

A groundbreaking topical gene therapy for 'butterfly skin disease' has transformed rare disease treatment. Suma Krishnan's innovative approach, applying genetic medicine directly to the skin, led to ...

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Anyone looking for evidence of genetic medicine’s ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...