All articles tagged with #rna therapy
Scientists are pursuing RNA-delivering gene therapies to disable a brake on heart-cell division in hopes of regenerating failing hearts; early animal data show improved heart function and a first-in-human trial began, but the field remains cautious due to uncertain evidence of true regeneration and a history of controversial retractions.
Columbia researchers engineered RNA-lipid nanoparticles that, when injected into skeletal muscle, make pro-ANP. This inactive molecule travels to the heart and is activated by the enzyme Corin to become active ANP, promoting blood vessel growth, reducing inflammation, and limiting scar tissue to help repair the heart after a heart attack. Using self-amplifying RNA, a single injection can sustain activity for about four weeks, improving heart function and reducing damage in multiple animal models, including delayed treatment scenarios. The approach avoids direct heart delivery, offering a potentially simpler therapy with plans for a phase‑one safety trial and potential applications to other organs.
Nature News & Views highlights Lei et al.’s method for engineering lipid nanoparticles that specifically deliver therapeutic RNA to pancreatic cell populations in animals, addressing liver-biased distribution and enabling new avenues for treating currently incurable pancreatic diseases.
A China-led international research team has discovered that xalnesiran, a small interfering RNA molecule, can potentially cure about one-third of hepatitis B patients when used alone or with an immunomodulator. This finding, published in The New England Journal of Medicine, marks a significant advancement in treating a virus that affects over 250 million people globally, with China having the highest number of cases. Current antiviral treatments can control but not eradicate the virus, making this discovery a promising step towards a cure.
Northwestern Medicine researchers have discovered a potential new approach to treating Alzheimer's disease by focusing on the balance of protective and toxic strands of short RNA in the brain. This fresh perspective could lead to the development of drugs that stabilize or delay the demise of protective RNA, potentially benefiting not only Alzheimer's patients but also those with other complex neurodegenerative diseases such as ALS or Parkinson's. The team is continuing their research in animal models and believes any new treatment would likely be given in combination with current therapies.