Title:Gene Therapy, A Potential Therapeutic Tool for Neurological and Neuropsychiatric Disorders: Applications, Challenges and Future Perspective
Volume: 23
Issue: 1
关键词:
基因治疗，神经退行性疾病，神经精神疾病，CRISPR-Cas9，病毒载体，基因。
摘要: Neurological and neuropsychiatric disorders are the main risks for the health care system,
exhibiting a huge socioeconomic load. The available range of pharmacotherapeutics mostly provides
palliative consequences and fails to treat such conditions. The molecular etiology of various neurological
and neuropsychiatric disorders is mostly associated with a change in genetic background, which
can be inherited/triggered by other environmental factors. To address such conditions, gene therapy is
considered a potential approach claiming a permanent cure of the disease primarily by deletion, silencing,
or edition of faulty genes and by insertion of healthier genes. In gene therapy, vectors (viral/nonvial)
play an important role in delivering the desired gene to a specific region of the brain. Targeted
gene therapy has unraveled opportunities for the treatment of many neurological and neuropsychiatric
disorders. For improved gene delivery, the current techniques mainly focus on designing a precise viral
vector, plasmid transfection, nanotechnology, microRNA, and in vivo clustered regulatory interspaced
short palindromic repeats (CRISPR)-based therapy. These latest techniques have great benefits
in treating predominant neurological and neurodevelopmental disorders, including Parkinson's disease,
Alzheimer's disease, and autism spectrum disorder, as well as rarer diseases. Nevertheless, all these
delivery methods have their limitations, including immunogenic reactions, off-target effects, and a deficiency
of effective biomarkers to appreciate the effectiveness of therapy. In this review, we present a
summary of the current methods in targeted gene delivery, followed by the limitations and future direction
of gene therapy for the cure of neurological and neuropsychiatric disorders.
