Article | January 22, 2024

Delivering The Promise Of Gene Therapy

Source: Battelle
GettyImages-597935610 cell and gene

Gene therapy has seen significant advancements in recent years, particularly with the development of CRISPR technology. Clinical trials for CRISPR-based therapies in humans are already underway, and it is expected that the first FDA-approved CRISPR therapy will be available by 2024.

However, the future of gene therapy relies on finding efficient and precise methods of delivering genetic material. Traditional viral delivery methods have limitations, such as high costs, low cargo capacity, prolonged expression, unintended immunity, and immunogenicity.

A potential solution lies in polymeric nanoparticles (PNPs) as a non-viral delivery method for gene therapy. PNPs have the advantage of carrying a larger payload than viral vectors, can be designed to target specific cell types, allow for repeat application, and naturally degrade once the intended cells are modified.

Researchers are currently developing a synthesis platform that incorporates tracking and machine learning capabilities to screen thousands of nanoparticles simultaneously, enabling the development of more effective drugs for patients at a faster rate. Examine how combining PNPs with the next generation of CRISPR-based gene therapies is increasing the potential for improved treatments.

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