Peptide Shuttle-Mediated Delivery for Brain Gene Therapies

被引:3
|
作者
Garcia, Josep [1 ]
Arranz-Gibert, Pol [1 ]
Sanchez-Navarro, Macarena [1 ]
Giralt, Ernest [1 ,2 ]
Teixido, Meritxell [1 ]
机构
[1] Barcelona Inst Sci & Technol BIST, Inst Res Biomed IRB Barcelona, Barcelona 08028, Spain
[2] Univ Barcelona, Dept Inorgan & Organ Chem, Barcelona 08028, Spain
关键词
Gene therapy; Genome editing; CRISPR; Viral vectors; Nanoparticles; Central nervous system; Drug delivery; peptide shuttles; RABIES VIRUS GLYCOPROTEIN; SMALL-INTERFERING RNA; PARKINSONS-DISEASE; IN-VITRO; HEPATOCELLULAR-CARCINOMA; LIPID NANOPARTICLES; INFECTIOUS DELIVERY; LENTIVIRAL VECTORS; GOLD NANOPARTICLES; RETROVIRAL VECTORS;
D O I
10.2174/1568026620666201023122903
中图分类号
R914 [药物化学];
学科分类号
100701 ;
摘要
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 side-effects. 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.
引用
收藏
页码:2945 / 2958
页数:14
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