Clinical gene therapy development for the central nervous system: Candidates and challenges for AAVs.

J Control Release

Department of Bioengineering, The University of Texas at Dallas, Richardson, TX 75080, USA; Department of Mechanical Engineering, The University of Texas at Dallas, Richardson, TX 75080, USA; Center for Advanced Pain Studies, The University of Texas at Dallas, Richardson, TX 75080, USA; Department of Surgery, The University of Texas at Southwestern Medical Center, Dallas, TX 75390, USA. Electronic address:

Published: May 2023

Many diseases affecting the central nervous system (CNS) are deadly but less understood, leading to impaired mental and motor capabilities and poor patient prospects. Gene therapy is a promising therapeutic modality for correcting many genetic disorders, expanding in breadth and scope with further advances. This review summarizes the candidate CNS disorders for gene therapy, mechanisms of gene therapy, and recent clinical advances and limitations of gene therapy in CNS disorders. We highlight that improving delivery across CNS barriers, safety, monitoring techniques, and multiplexing therapies are predominant factors in advancing long-term outcomes from gene therapy.

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Source
http://dx.doi.org/10.1016/j.jconrel.2023.04.009DOI Listing

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