Artistic rendering of gene editing reagents — mRNA (red) and DNA (green and yellow) constructs — being packaged into a lipid nanoparticle (blue). UCLA researchers have developed a lipid ...

Could next-generation gene editing mean a cure for cystic fibrosis (CF)? There's been a lot of advancement on the disease in the last decade, but there are still those living with the disease who get ...

Congenital bilateral absence of the vas deferens (CBAVD) is a recognised cause of male infertility most frequently linked to mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) ...

MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, ...

However, people with other mutations in the CFTR gene, including those with variants that result in no proteins being made at all, cannot benefit from the drug. “Therefore, interest is being placed on ...

Cystic fibrosis (CF) is a common genetic disorders that has been well studied. Researchers have identified CF-causing mutations in a gene called CFTR, which encodes for an ion channel. The genetic ...

This photo provided by Emilys Entourage in April 2025 shows Emily Kramer-Golinkoff, who has cystic fibrosis caused by a rare genetic mutation, during a trip to Maine. (Emilys Entourage via AP) ...

MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, ...

The FDA approved a once-daily oral combination of vanzacaftor/tezacaftor/deutivacaftor (Alyftrek) for cystic fibrosis (CF) in adults and children 6 years and up ...

Researchers have demonstrated that lipid nanoparticles can package and deliver an entire therapeutic gene along with gene-editing machinery - a combination of large, complex components that non-viral ...