Gene Therapy Delivery

Public Domain, https://commons.wikimedia.org/w/index.php?curid=488192

By Nandiyanto - Own work, Public Domain, https://commons.wikimedia.org/w/index.php?curid=5947359

There is much excitement about the potential of gene therapy, particularly using the CRISPR method.  What many people do not understand is that a major bottleneck for gene therapy is not the therapy method itself but its delivery to the target cells.  What is needed is a delivery system that is efficient, specific (to the target if possible, unless the gene therapy itself is specific and then delivery can be more general), and safe (little or no side effects).  Viral delivery is efficient, but can have severe side effects, and has caused death.  Nanoparticles are an alternative with much potential, but require further development, including increasing efficiency.  The delivery problem needs to be solved before the potential of gene therapy can be fully realized.  Abstract:

Clustered regularly interspaced short palindromic repeat-CRISPR-associated protein (CRISPR-Cas) systems, found in nature as microbial adaptive immune systems, have been repurposed into an important tool in biological engineering and genome editing, providing a programmable platform for precision gene targeting. These tools have immense promise as therapeutics that could potentially correct disease-causing mutations. However, CRISPR-Cas gene editing components must be transported directly to the nucleus of targeted cells to exert a therapeutic effect. Thus, efficient methods of delivery will be critical to the success of therapeutic genome editing applications. Here, we review current strategies available for in vivo delivery of CRISPR-Cas gene editing components and outline challenges that need to be addressed before this powerful tool can be deployed in the clinic.