Gene-Based Therapeutics for Parkinson's Disease

被引:5
|
作者
Shalaby, Karim E. [1 ,2 ]
El-Agnaf, Omar M. A. [1 ,2 ]
机构
[1] Hamad Bin Khalifa Univ HBKU, Qatar Fdn, Coll Hlth & Life Sci CHLS, Biol & Biomed Sci Div, Doha 34110, Qatar
[2] Hamad Bin Khalifa Univ HBKU, Qatar Fdn, Neurol Disorders Res Ctr, Qatar Biomed Res Inst QBRI, Doha 34110, Qatar
关键词
neurodegeneration; Parkinson's disease; gene therapy; AMINO-ACID DECARBOXYLASE; ALPHA-SYNUCLEIN GENE; CHAPERONE-MEDIATED AUTOPHAGY; CONVECTION-ENHANCED DELIVERY; TYROSINE-HYDROXYLASE; OPEN-LABEL; LEWY BODY; PHASE-I; GLUCOCEREBROSIDASE MUTATIONS; NEUROTROPHIC FACTOR;
D O I
10.3390/biomedicines10081790
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Parkinson's disease (PD) is a complex multifactorial disorder that is not yet fully surmised, and it is only when such a disease is tackled on multiple levels simultaneously that we should expect to see fruitful results. Gene therapy is a modern medical practice that theoretically and, so far, practically, has demonstrated its capability in joining the battle against PD and other complex disorders on most if not all fronts. This review discusses how gene therapy can efficiently replace current forms of therapy such as drugs, personalized medicine or invasive surgery. Furthermore, we discuss the importance of enhancing delivery techniques to increase the level of transduction and control of gene expression or tissue specificity. Importantly, the results of current trials establish the safety, efficacy and applicability of gene therapy for PD. Gene therapy's variety of potential in interfering with PD's pathology by improving basal ganglial circuitry, enhancing dopamine synthesis, delivering neuroprotection or preventing neurodegeneration may one day achieve symptomatic benefit, disease modification and eradication.
引用
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页数:18
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