Scientists studying aging may be taking an important step toward understanding how to preserve the body's cells as they grow ...
Cardiovascular diseases are the leading cause of death globally, with cardiac arrhythmias contributing substantially to this burden. Gene therapy, which directly targets the underlying disease ...
A new gene therapy appears to be safe in patients diagnosed with Friedreich ataxia cardiomyopathy, a progressive and fatal ...
Discusses Updated Phase 1/2 Data for NGN-401 Gene Therapy in Rett Syndrome June 29, 2026 8:00 AM EDTCompany ParticipantsLina ...
Treatment in which replacement gene placed in cold virus 'container' to be tested on 250 patients ...
Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...
Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...
Although promising, gene and cell therapies can pose genotoxic risks that complicate clinical application. We describe the molecular basis of these risks, discuss tools to assess genotoxicity and ...
Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...
Quality by design is critical for cell and gene therapy manufacturing where process controls directly influence product ...
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...
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