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
收藏
页数:18
相关论文
共 50 条
  • [1] Gene-based Therapies in Parkinson's Disease
    Allen, Patricia J.
    Feigin, Andrew
    [J]. NEUROTHERAPEUTICS, 2014, 11 (01) : 60 - 67
  • [2] Gene-based Therapies in Parkinson’s Disease
    Patricia J. Allen
    Andrew Feigin
    [J]. Neurotherapeutics, 2014, 11 : 60 - 67
  • [3] Gene-Based Therapeutics for Acquired Retinal Disease: Opportunities and Progress
    Tan, Tien-En
    Fenner, Beau James
    Barathi, Veluchamy Amutha
    Tun, Sai Bo Bo
    Wey, Yeo Sia
    Tsai, Andrew Shih Hsiang
    Su, Xinyi
    Lee, Shu Yen
    Cheung, Chui Ming Gemmy
    Wong, Tien Yin
    Mehta, Jodhbir Singh
    Teo, Kelvin Yi Chong
    [J]. FRONTIERS IN GENETICS, 2021, 12
  • [4] The Future of Targeted Gene-Based Treatment Strategies and Biomarkers in Parkinson's Disease
    Polissidis, Alexia
    Petropoulou-Vathi, Lilian
    Nakos-Bimpos, Modestos
    Rideout, Hardy J.
    [J]. BIOMOLECULES, 2020, 10 (06) : 1 - 32
  • [5] Gene Editing and Gene-Based Therapeutics for Cardiomyopathies
    Ohiri, Joyce C.
    McNally, Elizabeth M.
    [J]. HEART FAILURE CLINICS, 2018, 14 (02) : 179 - 188
  • [6] Gene-Based Therapeutics for Inherited Retinal Diseases
    Fenner, Beau J.
    Tan, Tien-En
    Barathi, Amutha Veluchamy
    Tun, Sai Bo Bo
    Yeo, Sia Wey
    Tsai, Andrew S. H.
    Lee, Shu Yen
    Cheung, Chui Ming Gemmy
    Chan, Choi Mun
    Mehta, Jodhbir S.
    Teo, Kelvin Y. C.
    [J]. FRONTIERS IN GENETICS, 2022, 12
  • [7] Nucleic Acid–Based Therapeutics for Parkinson’s Disease
    Masayuki Nakamori
    Eunsung Junn
    Hideki Mochizuki
    M. Maral Mouradian
    [J]. Neurotherapeutics, 2019, 16 : 287 - 298
  • [8] Cell-based Therapeutics for Parkinson's Disease
    Sanchez-Pernaute, Rosario
    [J]. METHODS AND FINDINGS IN EXPERIMENTAL AND CLINICAL PHARMACOLOGY, 2008, 30 : 9 - 10
  • [9] Gene-based approaches toward Friedreich ataxia therapeutics
    Hebert, M. D.
    Whittom, A. A.
    [J]. CELLULAR AND MOLECULAR LIFE SCIENCES, 2007, 64 (23) : 3034 - 3043
  • [10] Gene-based approaches toward Friedreich ataxia therapeutics
    M. D. Hebert
    A. A. Whittom
    [J]. Cellular and Molecular Life Sciences, 2007, 64 : 3034 - 3043