Next-generation cell therapies are reshaping therapeutics by precisely engineering living cells for both ex vivo and in vivo applications. Cutting-edge tools—including Cas enzymes (Cas9, Cas12a), base ...

Northwestern scientists have developed a new nanostructure that supercharges CRISPR’s ability to safely and efficiently enter cells, potentially unlocking its full power to treat genetic diseases. By ...

Acuitas’ LNP enabled delivery of three doses of a personalized CRISPR therapy with no adverse events to an infant patient with severe urea cycle disorder Athebody® DARPin-conjugated LNP resulted in ...

CRISPR has the power to correct genetic mutations, but current delivery methods are either unsafe or inefficient, keeping the technology from reaching its full medical potential. With the power to ...

CRISPR gene editing machinery holds the potential to transform the treatment of numerous diseases, but it requires effective delivery systems to get into tissues and ...

CRISPR gene editing has revolutionized the field of molecular biology, offering precise, efficient, and versatile tools for genome modification. The technology has rapidly evolved beyond the original ...

Lipid nanoparticles (LNPs) act as carriers for mRNA and CRISPR payloads across a wide range of therapeutic applications, from cancer to inflammatory and genetic diseases. The same delivery system used ...

Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for Duchenne muscular dystrophy (DMD) treatment. While adeno-associated virus ...