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Current Topics in Medicinal Chemistry


ISSN (Print): 1568-0266
ISSN (Online): 1873-4294

Review Article

Peptide Shuttle-Mediated Delivery for Brain Gene Therapies

Author(s): Josep Garcia, Pol Arranz-Gibert, Macarena Sánchez-Navarro, Ernest Giralt and Meritxell Teixidó*

Volume 20 , Issue 32 , 2020

Page: [2945 - 2958] Pages: 14

DOI: 10.2174/1568026620666201023122903

Price: $65


The manipulation of an individual’s genetic information to treat a disease has revolutionized the biomedicine field. Despite the promise of gene therapy, this treatment can have long-term sideeffects. Efforts in the field and recent discoveries have already led to several improvements, including efficient gene delivery and transfer, as well as inpatient safety. Several studies to treat a wide range of pathologies-such as cancer or monogenic diseases- are currently being conducted. Here we provide a broad overview of methodologies available for gene therapy, placing a strong emphasis on treatments for central nervous system diseases. Finally, we give a perspective on current delivery strategies to treat such diseases, with a special focus on systems that use peptides as delivery vectors.

Keywords: Gene therapy, Genome editing, CRISPR, Viral vectors, Nanoparticles, Central nervous system, Drug delivery, Peptide shuttles.

Graphical Abstract
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