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
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