Review
Biochemistry & Molecular Biology
Karim E. Shalaby, Omar M. A. El-Agnaf
Summary: Gene therapy has the potential to replace current treatments for Parkinson's disease and has been shown to be safe and effective in current trials.
Review
Biochemistry & Molecular Biology
Keelan Jagaran, Moganavelli Singh
Summary: Neurodegenerative disorders result in the gradual degeneration of axons and neurons in the central nervous system, causing major disruptions in patients' lives. Current treatments are only palliative, highlighting the need for a therapeutic strategy targeting the root cause of the diseases. The synergistic use of gene therapy and nanomedicine shows promise in effectively treating these diseases by targeting the causative mutated genes.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Genetics & Heredity
Jannik Prasuhn, Norbert Brueggemann
Summary: Mitochondrial dysfunction plays a crucial role in the pathophysiology of Parkinsonian disorders and may serve as a target for gene therapies. Overcoming key challenges is essential for the successful implementation of this treatment strategy.
Article
Clinical Neurology
Jean-Christophe Corvol, Jean-Philippe Azulay, Bjorn Bosse, Yves Dauvilliers, Luc Defebvre, Fabian Klostermann, Norbert Kovacs, David Maltete, William G. Ondo, Rajesh Pahwa, Werner Rein, Stephane Thobois, Martin Valis, Aleksandar Videnovic, Olivier Rascol
Summary: The study of THN102 in PD patients with EDS showed that the 200/2 dose demonstrated efficacy and good tolerability, supporting further development. On the other hand, the 200/18 dose did not show significant improvement.
MOVEMENT DISORDERS
(2022)
Article
Clinical Neurology
James C. Mamaril-Davis, Adrian W. Gelb, Paul S. Larson
Summary: A systematic review and meta-analysis found that patients in the sham surgery control group of clinical trials for Parkinson's disease have a low risk of procedure-related adverse events and fewer complications compared to patients in the experimental groups.
MOVEMENT DISORDERS
(2023)
Review
Biochemistry & Molecular Biology
Sri Jayanti, Rita Moretti, Claudio Tiribelli, Silvia Gazzin
Summary: With the increase in life expectancy, the prevalence of Parkinson's disease is expected to rise, but the diagnosis still relies on clinical symptoms. Current treatments mainly focus on alleviating symptoms without stopping or slowing down disease progression. Therefore, exploring the therapeutic potential of bilirubin in PD is crucial for the development of disease-modifying treatments.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Biochemistry & Molecular Biology
David Arango, Amaury Bittar, Natalia P. Esmeral, Camila Ocasion, Carolina Munoz-Camargo, Juan C. Cruz, Luis H. Reyes, Natasha I. Bloch
Summary: CRISPR is a popular gene-editing technique with potential applications in researching and treating neurodegenerative diseases, particularly Parkinson's disease. Gene therapy could offer new treatment alternatives and have significant potential in future research.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Engineering, Biomedical
Yifei Gao, Yuxue Cheng, Jiapeng Chen, Danmin Lin, Chao Liu, Ling-Kun Zhang, Liang Yin, Runcai Yang, Yan-Qing Guan
Summary: In this study, MgOp@PPLP nanoparticles were developed to overcome the blood-brain barrier and be used for gene therapy and antioxidant therapy in Parkinson's disease. These nanoparticles showed good neuroprotective effects in PD models.
ADVANCED HEALTHCARE MATERIALS
(2022)
Review
Clinical Neurology
Sindhu Menon, Sabrina Armstrong, Amir Hamzeh, Naomi P. Visanji, Sergio Pablo Sardi, Anurag Tandon
Summary: Therapeutic targeting of alpha-Synuclein shows promising treatment strategies to limit its pathogenic effects in neurodegenerative diseases. Different approaches include removing aggregated asyn, modulating misfolding kinetics, lowering gene expression, and promoting degradation pathways. Technological advances, such as combining ultrasound with microbubbles, aim to enhance brain delivery of anti-asyn biologics.
FRONTIERS IN NEUROLOGY
(2022)
Review
Cell Biology
Hamed Mirzaei, Saman Sedighi, Ebrahim Kouchaki, Erfaneh Barati, Ehsan Dadgostar, Michael Aschner, Omid Reza Tamtaji
Summary: Research has shown that probiotics can improve symptoms in patients with Parkinson's disease, and mediate pharmacological effects by targeting cellular and molecular processes.
CELLULAR AND MOLECULAR NEUROBIOLOGY
(2022)
Article
Biotechnology & Applied Microbiology
Feiyang Li, Jun Wu, Daiye Li, Liuzhi Hao, Yanqun Li, Dan Yi, Kelvin W. K. Yeung, Di Chen, William W. Lu, Haobo Pan, Tak Man Wong, Xiaoli Zhao
Summary: A stem cell-mediated gene therapy strategy was developed in this study, where genetically engineered mesenchymal stem cells were used to produce exosomes with enhanced bone regeneration potency. These engineered exosomes exhibited homing ability to the injured site and showed potential clinical applications.
JOURNAL OF NANOBIOTECHNOLOGY
(2022)
Review
Surgery
Stephanie N. Serva, Jacob Bernstein, John A. Thompson, Drew S. Kern, Steven G. Ojemann
Summary: This review provides an overview of currently available therapies for advanced Parkinson's disease (PD), including pharmacological management and advanced therapies, as well as discussing novel therapies currently being tested in clinical trials. It aims to equip healthcare providers with updated information on advanced therapies for PD in order to better advise patients on treatment options.
FRONTIERS IN SURGERY
(2022)
Article
Pharmacology & Pharmacy
Chung-Yin Lin, Ching-Yun Huang, Chiung-Mei Chen, Hao-Li Liu
Summary: In this study, a focused ultrasound-targeted microbubble destruction (UTMD)-mediated Parkinson's disease (PD) model was developed to generate the disease phenotype via gene delivery. The UTMD-based model mimicked the rapid development of PD and proved valuable in assessing specific aspects of PD pathogenesis.
Review
Chemistry, Multidisciplinary
Junguang Wu, Xuejing Cui, Pu Chun Ke, Monika Mortimer, Xiaoyu Wang, Lin Bao, Chunying Chen
Summary: Parkinson's disease is a complex multi-factorial disease with no cure, only mitigative therapies available at present. The development of nanomedicine has provided new opportunities for finding neuroprotective and neurorestorative therapies for PD. By exploring various mechanisms underlying PD pathology and discussing nanomaterial-based treatment strategies, there is potential for targeted therapies to be developed in the future.
Article
Clinical Neurology
Yaping Chu, Jeffrey H. Kordower
Summary: This study performed post-mortem studies on two patients with advanced Parkinson's disease to verify the long-term effects of the trophic factor neurturin. The results showed persistent neurturin expression and bioactivity following gene delivery, which could enhance dopamine neuron function.
MOVEMENT DISORDERS
(2023)
